Research News

New insights into Zika's microcephaly link, similarity to dengue (Dec 13, 2018)
New insights into how dengue and Zika viruses cause disease reveal shared and virus-specific mechanisms. An international, multi-institutional team led by researchers of the University of California, San Francisco and Baylor College of Medicine report in the journal Cell that these viruses counteract a human and mosquito immune defense mechanism and hijack specific host proteins for virus replication. They also discovered that Zika virus causes microcephaly in fruit flies by disrupting the function of ANKLE2, a protein involved in brain development both in flies and humans. These findings open new avenues to design therapeutic strategies to combat these widespread and severe infectious diseases.


Dr. Huda Zoghbi elected as a fellow of the National Academy of Inventors (Dec 13, 2018)
Dr. Huda Zoghbi has been elected as a fellow of the National Academy of Inventors this week. Drs. Mary K. EstesBert O’Malley and Huda Zoghbi of Baylor College of Medicine were three of the 148 renowned academic inventors who received the distinction this week.


Understanding formation of new neurons in adult brains (Dec 10, 2018)
A team of researchers at Baylor College of Medicine, the Texas Heart Institute and Texas Children’s Hospital has developed a powerful new approach to understand the formation of new neurons in the mammalian adult brain. Published in the journal Cell Reports, this work opens exciting new pathways that can be further developed to repair malfunctioning brain circuits.


Loss of SYNGAP1 function results in abnormalities in sensory processing (Nov 19, 2018)
In addition to cognitive defects, neurodevelopmental disorders are often associated with deficits in sensory perception and processing. However, very little is known about the biological mechanisms that underlie these sensory impairments.In a study published in Nature Neuroscience, one of the co-authors, Dr. Jimmy L. Holder Jr., neurologist and director of the new SYNGAP1 Center of Excellence at Texas Children’s Hospitalinvestigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s and assistant professor at Baylor College of Medicine, analyzed retrospective clinical data of SYNGAP1 patients from a registry maintained by Bridge the Gap – SYNGAP1 Education and Research Foundation.


Sudden death of a loved one is one of the common causes of reduced academic performance among youth (Nov 14, 2018)
A recent study led by Dr. Julie Kaplow, director of Trauma and Grief Center at Texas Children’s Hospital/Baylor College of Medicine finds sudden death of a loved one (e.g. parents, grandparents, close friend or relatives) is a common traumatic event experienced by teenagers and can have a profound negative impact on their academic performance and functioning. 


Sheehan helps design trial network for sickle cell disease (Nov 12, 2018)
Dr. Vivien Sheehan, assistant professor of pediatrics - hematology/oncology at Baylor and Texas Children's Cancer and Hematology Centers, has assisted in the design of the American Society of Hematology Sickle Cell Disease Clinical Trial Network to advance development of new and more effective therapeutics for individuals with sickle cell disease.


Dr. Sharon Plon's team develop a novel computational tool to annotate microRNA (Nov 12, 2018)
A graduate student in Dr. Plon's lab, Ninad Oak has developed a novel computational tool called ADmiRE, which stands for Annotative Database for miRNA Elements. ADmiRE extensively annotates human microRNA variants to determine which ones are likely to contribute to or cause disease.


Evenings with Genetics offers update on rare Angelman Syndrome (Nov 7, 2018)
The next installment of Evenings with Genetics on Tuesday, Nov. 13, will focus on Angelman Syndrome, with expert speakers providing an update on treatment and research efforts for the rare neurogenetic disease. Dr. Carlos Bacino, professor of molecular and human genetics at Baylor and part of the newly-formed Angelman Syndrome Clinic at Texas Children’s Hospital, and Dr. Arthur Beaudet, the Henry and Emma Meyer Chair and Professor in Molecular and Human Genetics at Baylor, will lead the discussion. They will be joined by a special guest speaker, Dr. Deborah Sukin, a parent advocate of a child with Angelman Syndrome.


Research reveals defective transport of lysosomal enzymes causes a type of Batten disease (Nov 7, 2018)
Neuronal Ceroid Lipofuscinoses (NCLs a.k.a. Batten disease) are a group of fatal neurodegenerative disorders that result from defective lysosomal function due to mutations in different CLN genes. A surprising discovery from the laboratory of Dr. Marco Sardiello, assistant professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital shows defective lysosomal biogenesis as the underlying cause of Neuronal Ceroid Lipofuscinoses 8 (NCL8).


Researchers find a potential new approach to control cancer growth (Oct 19, 2018)
A team of researchers at Baylor College of Medicine and Texas Children’s Hospital has revealed a connection between mTORC1 and Src, two proteins known to be hyperactive in cancer. The study, published in the journal Nature Communications, shows that Src is necessary and sufficient to activate mTORC1 and offers the possibility to develop novel approaches to control cancer growth.


Hispanic children with acute lymphoblastic leukemia at higher risk for neurotoxicity (Oct 12, 2018)
Michael E. Scheurer, PhD, MPH, associate professor of pediatrics at Baylor College of Medicine and director of the Epidemiology Center at Texas Children’s Cancer Center, and colleagues analyzed medical records of 280 pediatric patients with newly diagnosed acute lymphoblastic leukemia (ALL).Hispanic children with ALL appeared more likely than non-Hispanic patients to develop neurotoxicity during methotrexate chemotherapy, according to study results.


Dr. Gaber earns mentorship award (Oct 12, 2018)
Dr. M. Waleed Gaber, associate professor of pediatrics (hematology/oncology) at Baylor College of Medicine and at TexasChildrens's Cancer and Hematology Centers, received the Scholar-in-Training Excellence in Mentorship Award from the Radiation Research Society for providing exceptional professional and personal mentoring.


Dr. Hari Krishna Yalamanchili receives the 2018 NRI Zoghbi Scholar Award (Oct 11, 2018)
Dr. Hari Krishna Yalamanchili, Ph.D., postdoctoral associate in the laboratory of Dr. Zhandong Liu, at Baylor College of Medicine, received the 2018 NRI Zoghbi Scholar Award, a special fund established by Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children's Hospital, to honor the many generations of trainees who have worked in her laboratory and to foster the next generation of successful scientists.


Undiagnosed Diseases Network finds 31 new syndromes (Oct 11, 2018)
Identifying genes responsible for rare or unknown disorders using traditional approaches can be extremely time-consuming and typically takes several years or decades. However, the Undiagnosed Diseases Network (UDN), a consortium of researchers including Baylor College of MedicineTexas Children’s HospitalStanford University and other institutions, has found 31 new syndromes and has been able to make diagnoses for 132 patients within two years of the group’s inception.


New high-dose radiation therapy available for pediatric liver cancer patients (Oct 5, 2018)
Pediatric liver cancer affects approximately 200 children each year in the United States. Treatment options are limited if the tumor cannot be completely removed with surgery. Although chemotherapy can be used, it often has limited efficacy and is associated with significant short and long term side effects. Thus, there is an urgent need to find therapeutic strategies to help children with liver tumors. A team of physician-scientists at Baylor College of Medicine and Texas Children’s Hospital are now using a new treatment option for pediatric patients with liver tumors, called Transarterial Radioembolization (TARE).


Research reveals a novel link between congenital cataracts and dynamin-binding protein (Oct 4, 2018)
Cataract, a condition in which the eye’s natural lens get clouded, is the most common cause of vision loss in older people and can be corrected by routine surgery. But congenital cataract, which occurs in infants and children, is particularly serious since it can inhibit visual development leading to permanent vision loss or impairment, which cannot be entirely reversed with cataract surgery. A new study has now found compelling evidence that links dynamin-binding protein (DNMBP) to congenital bilateral cataract and severe vision loss. 


SYNGAP1 Center of Excellence at Texas Children’s Hospital receives research grant (Oct 3, 2018)
Jimmy Holder Jr., M.D./Ph.D., a pediatric neurologist and scientist at the  Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and assistant professor at Baylor College of Medicine, was awarded a $25,000 research grant from the Bridge the Gap – SYNGAP Education and Research Foundation


Dr. Hsiao-Tuan Chao awarded NIH Director's High-Risk, High-Reward grant (October 2, 2018)
Dr. Hsiao-Tuan Chao, instructor of pediatrics at Baylor College of Medicine and a child neurologist and postdoctoral researcher at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, has been awarded the National Institutes of Health Director’s Early Independence Award from the High-Risk, High-Reward Research program


Texas Children's hematologist, Dr. Vivien Sheehan, develops treatment for Sickle Cell disease (September 27, 2018)
Sickle cell disease affects more than 100,000 Americans and millions more worldwide. This red blood cell disorder can be treated, but a widely available cure has yet to be found. Texas Children’s hematologist and Assistant Professor of Pediatrics Dr. Vivien Sheehan has recently developed a new sickle cell treatment based on her laboratory research. Sheehan has found that metformin, a commonly used medication for diabetes, has the ability to induce fetal hemoglobin in developing red blood cells.


Drs. Philip Lupo and Karen Rabin receive NIH grant to study the link between Down syndrome and Acute Lymphoblastic Leukemia (ALL) (September 25, 2018)
Children with Down syndrome are about 20 times more likely to develop acute lymphoblastic leukemia (ALL) than other children. To better understand this predisposition for cancer, a team of researchers from Baylor College of Medicine, Emory University and St. Jude Children’s Research Hospital have been awarded a grant from theGabriella Miller Kids First Pediatric Research Program (Kids First) to sequence the genomes of more than 2,000 individuals with Down syndrome.


In the phase II, UDN expands to add five new clinical sites, a new metabolomics core and new model organism core (September 24, 2018)
NIH has given many new grants to academic medical centers across the nation to improve and accelerate the diagnosis of rare and undiagnosed The new awards are part of  the second phase of the National Institutes of Health’s Undiagnosed Diseases Network (UDN). 


CPRIT awards $6 million to find new biomarkers and therapies for childhood liver cancers (September 19, 2018)
Texas Children’s Hospital and Baylor College of Medicine researchers received a $6 million research grant from the Cancer Prevention and Research Institute of Texas to find new predictive biomarkers and therapies for high-risk pediatric liver cancers. 


Texas Children’s named Clinical Research Center of Excellence for CDKL5 disorders (September 10, 2018)
Texas Children’s Hospital has been recognized as a Clinical Research Center of Excellence (COE) for CDKL5 Deficiency Disorder and is one of only five pediatric institutions in the country to receive this honor.


Long genes are not preferentially altered in Rett and MeCP2 duplication syndromes (September 7, 2018)
Many recent large-scale transcriptome studies have shown that long genes (>100 kb) are preferentially dysregulated in neurodevelopmental disorders. A recent study from the laboratory of Dr. Zhandong Liu, associate professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital shows that expression of long genes is not preferentially altered in MeCP2 syndromes.


New histiocytotic disorder insights may improve therapies (August 30, 2018)
In a review published in New England Journal of Medicinephysician-scientists from Baylor College of Medicine, Texas Children’s Cancer and Hematology Centers and the Icahn School of Medicine review the history of LCH and the recent developments that will help propel the treatment for patients with LCH into the realm of personalized medicine.


Two neuronal lineages in the hindbrain act together to ‘jumpstart’ rhythmic breathing at birth (August 28, 2018)
A study published in eLife from the Zoghbi lab has found two neuronal lineages in the hindbrain coordinate and relay the changes in oxygen and carbondioxide levels to the rhythmogenic neurons in the central respiratory circuit to establish and maintain optimal breathing rhythms, which are especially critical for the survival of a newborn.


Southwest Pediatric Device Consortium at Texas Children’s Hospital and Baylor College of Medicine receives $6.75M FDA P50 grant to expand development of medical devices for children (August 24, 2018)
The Southwest National Pediatric Device Consortium (SWPDC), anchored by Texas Children’s Hospital and Baylor College of Medicine, recently received a prestigious P50 grant from the U.S. Food & Drug Administration (FDA). The five-year, $6.75M grant will begin on Sept. 1


Xue lab develops a better optogenetic tool to study neuronal function (August 20, 2018)
The scientists in the laboratory of Dr. Mingshan Xue, assistant professor at Baylor College of Medicine and an investigator at the Cain Foundation Laboratories and the  Jan and Dan Duncan Neurological Research Institute atTexas Children's Hospital, have modified an existing light-sensitive tool to produce fewer undesired effects, making it a better tool to study neuronal function. 


Texas Children’s leads effort to get lifesaving drug approved by FDA (August 20, 2018)
Thanks to the more than 10 years of dedication and hard work of neonatologists and neonatal nutrition experts at Texas Children’s, Boston Children’s and the University of California at Los Angeles (UCLA) hospitals, premature babies with liver disease now have better access to a potentially lifesaving drug.


Four pediatrics faculty receive the 2018 research mentorship awards (August 14, 2018)
The following Baylor College of Medicine/Texas Children’s Hospital faculty affiliated to the department of pediatrics were selected for the 2018 research mentorship awards for providing outstanding research guidance to medical, graduate students, and postdoctoral trainees - Andrea Cruz, MD MPH, associate professor of pediatrics (Emergency Medicine section), Richard Kellermayer, MD, associate professor of pediatrics (Gastroenterology section), Stacey Berg, MD, professor of pediatrics (Texas Children’s Cancer and Hematology centers) and Deborah Thompson, PhD, RD, professor of pediatrics (Children's Nutrition Research Center).


Srivaths named to blood disorders foundation (August 3, 2018)
Dr. Lakshmi Srivaths, professor in the department of pediatrics and co-director of Young Women's Bleeding Disorders Clinic, has been appointed as chair of the education/advocacy subcommittee for the national Foundation for Women and Girls with Blood Disorders. The chair will be in charge of the subcommittees organization and ongoing activities, which include creating education for clinic staff, referring providers, providers outside of primary institution and organized/informal education opportunities.


Fruit flies help UDN researchers uncover new neurological disorder in seven patients (July 27, 2018)
Spontaneously arising mutations in a single copy of the IRF2BPL gene are associated with a previously undiagnosed neurological disorder in seven unrelated individuals, reports a recent collaborative study. This project was initiated when the corresponding author of this study, Dr. Loren D.M. Pena, associate professor of pediatrics; and Dr. Vandana Shashi, professor of pediatrics, both at the Duke Clinical Site of the Undiagnosed Disease Network (UDN); connected Dr. Hugo Bellen, professor of molecular and human genetics and neuroscience at Baylor College of Medicine, an investigator at the Howard Hughes Medical Institute and the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital to a patient who presented with a severe neurological condition without a diagnosis.


St. Baldrick's Foundation awards seven faculty with pediatric cancer research grants (July 20, 2018)
The St. Baldrick’s Foundation, a volunteer-powered charity dedicated to raising money for childhood cancer research, has announced its 2018 summer grant recipients, including seven from Baylor College of Medicine and Texas Children’s Cancer and Hematology Centers.


The Teratology Society honors Dr. Lupo (July 17, 2018)
Dr. Philip Lupo, PhD, MPH, received the 2018 F. Clarke Fraser New Investigator Award from the Teratology Society, recognizing an early career scientist who has established a successful independent research career.


Dr. Judith Margolin receives a special biennial Founder's award from the Lymphangiomatosis and Gorham's Disease Alliance (July 15, 2018)
Dr. Judith Margolin, Co-Director of the Pediatric Hematology-Oncology Vascular Anomalies Program at Texas Children's receives the 2017-2018 recipient of the special biennial Founder's award from the Lymphangiomatosis and Gorham's Disease Alliance for her extraordinary professionalism, critical care, treatment and support of patients and their families affected by rare lymphatic disorders.


Pediatric neurosurgeon, Dr. Sandi Lam, develops a novel endoscopic procedure to treat intractable seizures (July 16, 2018)
Dr. Sandi Lam, pediatric neurosurgeon at Texas Children's Neuroscience Center, performed a novel endoscopic hemispherotomy as a surgical treatment to treat intractable seizures that has significantly reduced seizure episodes.


Dr. Hsiao-Tuan Chao receives a Child Neurology Foundation grant to study pediatric epilepsy and intellectual disability (July 3, 2018)
Dr. Hsiao-Tuan Chao, MD/PhD, clinical child neurologist and associate director of the child neurology residency program at Texas Children’s Hospital was awarded the Child Neurology Foundation’s 2018 Pediatric Epilepsy Research Foundation Grant. 


Mutations in gene TRAF7 associated with multisystem disorder (June 28, 2018)
A group of seven patients presenting with a similar disorder of unknown origin now know of a possible genetic root of their condition. 


Rice University, Texas Children's Hospital and Baylor College of Medicine win major NIH grant to study discrete subaortic stenosis (June 27, 2018)
Scientists at Rice University, Texas Children's Hospital and Baylor College of Medicine have won a prestigious National Institutes of Health grant to study the dynamic processes and cellular players linked to discrete subaortic stenosis (DSS), a congenital heart disease. 


A new integrative approach finds viable therapeutic targets for Huntington's disease (June 21, 2018)
When a complex system goes awry and multiple parts are affected, how do you figure out which changes are causative and which are merely correlative? A team of scientists led by Dr. Juan Botas, professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute have developed a way to sort through the thousands of genetic alterations that accumulate in human brains affected by neurological disease. 


A simple sugar delays neurodegeneration in lysosomal storage disorders (June 20, 2018)
A new study from the laboratory of Dr. Marco Sardiello, assistant professor in Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, has found oral administration of trehalose, a simple sugar, can alleviate the neurological symptoms associated with the deficiency of a lysosomal enzyme.


Ceramides may play a role in early-onset Parkinson’s disease (June 18, 2018)
The researchers in the laboratory of Dr. Hugo Bellen, professor at Baylor College of Medicine and an investigator at the Howard Hughes Medical Institute and at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, have found ceramides, a family of lipid molecules found within cell membranes, play an important role in an early-onset form of Parkinson’s disease. The study was published in the journal Cell Metabolism.


Dr. Zhandong Liu receives distinguished service award (June 18, 2018)
Dr. Zhandong Liu, assistant professor at Baylor College of Medicine and researcher at the Jan and Dan Duncan Neurological Research Institute and member of the Dan L Duncan Comprehensive Cancer Center, has received a distinguished service award from the International Association for Intelligent Biology and Medicine (IAIBM).


Dr. Mingshan Xue awarded 2018 McKnight Scholar Award (June 14, 2018)
Dr. Mingshan Xue, assistant professor of neuroscience at Baylor College of Medicine and a researcher with the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, has been awarded the prestigious 2018 McKnight Scholar Award.  


Dr. Hsiao-Tuan Chao receives the prestigious Burroughs Wellcome Fund Career Award (June 11, 2018)
Dr. Hsiao-Tuan Chao, a child neurologist and postdoctoral researcher at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, was awarded the highly-competitive 2018 Burroughs Wellcome Fund Career Award for Medical Scientists.


Tau aggregates activate “jumping genes” in Alzheimer’s disease (June 8, 2018)
A recent study published in Cell Reports has found a novel mechanism by which Tau protein aggregates might contribute to neurodegeneration. In this collaborative study, researchers found numerous transposons (a.k.a jumping genes) were activated in the brains of deceased subjects affected by Alzheimer’s disease (AD). Further, using fruit fly models of AD they showed Tau protein is sufficient to activate transposons contributing to genomic instability that likely, triggers neurodegeneration.


Fruit fly study links TBX2 to a new Mendelian disorder (May 23, 2018)
An interdisciplinary team led by Dr. Shinya Yamamoto has found a link between TBX2, a member of the T-box family of transcription factors, to a novel disorder that mainly affects the cardiac, skeletal, immune and endocrine systems. The study appears in the journal Human Molecular Genetics.


Texas Children's research trainees receive national awards (May 17, 2018)
The Society for Pediatric Research (SPR) recently gave two Texas Children’s trainees national research awards for their outstanding research on kidney disease at this year’s annual Pediatric Academic Societies’ SPR meeting.


Texas Children's surgeons use state-of-the-art real-time 3-D visualization technology to streamline surgeries (May 9, 2018)
In March, pediatric surgical specialists at Texas Children’s Hospital have used the Orbeye for pediatric plastic, urology and congenital heart surgeries. Orbeye is a new freestanding videomicroscope that displays high-resolution 3-D imaging of surgical procedures on an expansive 55-inch 4K monitor with stunning clarity. It’s like bringing the IMAX movie theater experience into the operating theater.


Fruit fly study identifies a new link to aortic aneurysms. (April 25, 2018)
An interdisciplinary team of researchers has identified a new gene linked to human aortic aneurysms. By combining comprehensive genetic studies in the fruit fly, dataset searches and analysis of diseased human aortic tissue, the researchers shed light on mechanisms not previously known to be associated with this devastating disease. The study appears in the journal Developmental Cell.


FDA approves everolimus for tuberous sclerosis-associated seizures, a treatment first discovered at Texas Children’s Hospital (April 20, 2018)
On April 10, 2018, the Food and Drug Administration (FDA) approved everolimus tablets for oral suspension (brand name: Afinitor Dispersz, Novartis Pharmaceuticals Corporation) as an adjunctive therapy to treat partial-onset refractory seizures in adults and children over 2 years of age with Tuberous Sclerosis Complex (TSC)


Dr. Peter Hotez and Dr. Huda Zoghbi elected as members of the American Academy of Arts and Sciences, one of nation's most prestigious honorary titles (April 18, 2018)
Dr. Peter J. Hotez, professor and dean of the National School of Tropical Medicine at Baylor College of Medicine, and Dr. Huda Y. Zoghbi, professor and Howard Hughes Medical Institute Investigator at Baylor, both also with Texas Children’s Hospital, have been elected as the newest members of the American Academy of Arts and Sciences, one of the nation’s most prestigious honorary titles.


Dasatinib is a safe and effective treatment for pediatric Chronic Myeloid Leukemia in chronic phase (CML-CP) (April 6, 2018)
In the largest prospective phase II trial till date of its kind, researchers report dasatinib to be safe and effective for children with CML-CP. 


Dr. Stephen Mack receives the First-Time, Tenure-Track Faculty Members Award from CPRIT (April 5, 2018)
The Cancer Prevention and Research Institute of Texas (CPRIT) has conferred the First-time, Tenure-Track Faculty Members Award on Dr. Stephen Mack to recruit him to Baylor College of Medicine from Cleveland Clinic.


The Bellen lab generates a large "CRIMIC" library, a versatile tool to accelerate biomedical discovery (March 30, 2018)
An exciting study published in eLife reports the generation of a large versatile library of fruit flies that can be used to perform efficient and elegant in vivo gene-specific manipulations.


Dr. David Nelson elected as the President of the American Society of Human Genetics (March 21, 2018)
David L. Nelson, Ph.D.,a Cullen Foundation Professor of Molecular and Human Genetics at the Baylor College of Medicine (NCM), Associate Director of the BCM Intellectual and Developmental Disabilities Research Center, and Director of the BCM Integrative Molecular and Biomedical Sciences Graduate Program and an investigator at the Jan and Dan Duncan Neurological Research Institute, has been elected as the 2018 President of the American Society of Human Genetics (ASHG). 


The Sardiello lab develops a new web tool to speed up biomedical discovery (March 12, 2018)
Researchers in the laboratory of Dr. Marco Sardiello, assistant professor at Baylor College of Medicine and investigator in the  Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, have developed a new web tool called Aminode that will help researchers analyze and predict proteins’ evolutionarily conserved regions (ECRs).


Dystonia Medical Research Foundation interviews Dr. Roy Sillitoe about his research in dystonia (March 9, 2018)
Dystonia Medical Research Foundation interviews Dr. Roy Sillitoe to learn how recent discoveries from his lab on a brain region called cerebellum can help dystonia patients. Watch the video


Research reveals mechanism that drives ataxia type 1 (March 9, 2018)
A group of researchers at Baylor College of Medicine, Texas Children’s Hospital and the University of Minnesota reports in the journal Neuron that polyQ-ATAXIN1 and a partner molecule called capicua form a complex that is essential for neurodegeneration. 


Molecular Medicine awards Ross Prize to Dr. Huda Zoghbi (Mar 8, 2018)
Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, Howard Hughes Medical Institute investigator and professor at Baylor College of Medicine, has been awarded the sixth annual Ross Prize in Molecular Medicine by The Feinstein Institute for Medical Research at Northwell Health.


National Cancer Institute awards Dr. Susan Blaney (Mar 6, 2018)
Dr. Susan Blaney, Professor and Executive Vice Chair for the Department of Pediatrics at Baylor College of Medicine and the Deputy Director of Texas Children’s Cancer Center, received a Director’s Service Award from the National Cancer Institute for her outstanding and dedicated service to the Institute and the Clinical Trials and Translational Research Advisory Committee from 2015 to 2017.


Research reveals path to ependymoma chemotherapy (Feb 28, 2018)
Ependymoma is a type of brain tumor that is resistant to chemotherapy.  Dr. Stephen Mack, assistant professor of pediatrics – oncology and new faculty member at Baylor College of Medicine and Texas Children’s Hospital, and colleagues have developed a framework for discovering targets in ependymomas, and other cancer that lack known genetic drivers, thereby also providing insights into treatment strategies. 


Texas Children's raises awareness for rare diseases (Feb 28, 2018)
This past Saturday, Feb. 24, hundreds of patients, family members, community advocates, health care providers and more joined Texas Children’s Hospital and Baylor College of Medicine to gather at The Health Museum in Houston, raising awareness for rare diseases in observance of the upcoming Rare Disease Day.


Automated detection of HFOs identify source of seizures in epilepsy patients (Feb 26, 2018)
Drs.
 Daniel Curry and Michael Quach from Texas Children’s Division of Neurology and Neurosurgery co-authored an insightful epilepsy study that was recently published in Brain, a Journal of Neurology.


Texas Children’s celebrates Blue Bird Circle’s 95th birthday and partnership (Feb 24, 2018)
Since 1998, the Blue Bird Circle has supported the Clinic for Pediatric Neurology at Texas Children’s with millions of dollars in grants, as well as thousands of volunteer hours. On February 6, Macey and 100 Blue Bird members including many of Texas Children’s employees, staff and leaders, attended a special luncheon at the Pavilion for Women to celebrate the Blue Bird Circle’s 95th birthday.


Changes in Pumilio1 levels linked to distinct neurological disorders (Feb 22, 2018)
In a collaborative study published in Cell, mutations that alter the protein levels of PUMILIO1 (PUM1) can cause a variety of neurological symptoms ranging from mild adult-onset ataxia to childhood-onset ataxia, intellectual disability, developmental delay and seizures. This is the first study to uncover a role for PUM1 in human brain function and to implicate it in two novel neurological syndromes.


Dr. Peter Hotez appointed to U.S. - Israel Binational Science Foundation Board of Governors (Feb 12, 2018)
Dr. Peter Hotez, dean of the National School of Tropical Medicine at Baylor College of Medicine and Texas Children’s Hospital Endowed Chair in Tropical Pediatrics, has been appointed as a representative to the U.S.-Israel Binational Science Foundation Board of Governors. The announcement was made today by the U.S. Department of State.


One little boy's DNA brings the promise of a cure for Rett syndrome girls (Jan 30, 2018)
Dr. Huda Zoghbi's lab has found some tantalizing clues by studying the DNA of a little boy, Henry Engel, who has a mutation in MeCP2 gene. These findings could one day help researchers to develop treatment for girls suffering from Rett syndome. Learn more


Dr. Huda Zoghbi receives Lebanon's highest honor by Lebanese President General Michel Aoun (Jan 23, 2018)
Dr. Huda Zoghbi, director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital,Howard Hughes Medical Institute investigator, the Ralph D. Feigin Endowed Chair and  professor at Baylor College of Medicine, was awarded Lebanon’s highest honor, the National Order of the Cedar, Knight grade, by Lebanese President General Michel Aoun.


Texas Children’s Hospital receives grant to continue developing a vaccine for Chagas disease (Jan 22, 2018)
Texas Children’s Hospital is excited to announce it received a $1.9 million grant from The Robert J. Kleberg Jr. and Helen C. Kleberg Foundation to support Texas Children’s Center for Vaccine Development (CVD), advancing the efforts to develop a therapeutic vaccine against Chagas disease.


Dr. Orange receives prestigious award for advancements in immunology research (Dec 14, 2017)
Dr. Jordan Orange, professor of pediatrics and chief of the section of Immunology, Allergy and Rheumatology at Baylor College of Medicine, and the director of the Center for Human Immunobiology at Texas Children’s, is the recipient of the 2018 Edith and Peter O’Donnell Award in Medicine from The Academy of Medicine, Engineering and Science of Texas (TAMEST).


Alex’s Lemonade Stand funds Phase 1 neuroblastoma trial (Dec 4, 2017)
Neuroblastoma is the most common solid tumor cancer in children and is challenging to treat regardless of existing therapies that are available. Dr. Andras Heczey, assistant professor of pediatrics - hematology and oncology, and Dr. Leonid Metelitsa, professor of pediatrics - oncology at Baylor College of Medicine, have received a $1.5 million grant from Alex’s Lemonade Stand Foundation to conduct a Phase 1, first-in-human clinical trial of a new form of immunotherapy to treat neuroblastoma using native and engineered properties of natural killer T-cells (NKTs).


Genetic mutations involved in childhood lysosomal storage disorders linked to increased risk of Parkinson's (Nov 17, 2017)
A new study by an international team of scientists led by Dr. Joshua Shulman, sheds new light on the genetic factors associated with Parkinson’s disease, a common neurodegenerative disorder.  


Charif Souki funds neurological research through $10 million gift to the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital (Nov 15, 2017)
Texas Children’s Hospital is excited to announce a $10 million commitment from Charif Souki to the Jan and Dan Duncan Neurological Research Institute (NRI) to fund the Huda Y. Zoghbi, M.D. Director’s Endowment. In honor of this gift, the NRI atrium will be named in Charif Souki’s honor and will now be called the Charif Souki Atrium.


Texas Children’s Cancer Center to offer state-of-the-art I-131 MIBG therapy (Nov 7, 2017)
As one of only a select number of pediatric hospitals in the U.S., Texas Children’s Cancer Center will now offer I-131 MIBG, a targeted nuclear medicine therapy that combines metaiodobenzylguanidine (MIBG) with radioactive iodine (I-131) to treat patients with high-risk neuroblastoma. This is the first pediatric treatment location in Houston designed specifically for this therapy and one of only a handful in the southern U.S. For more information about MIBG therapy at Texas Children’s Cancer Center, visit the Neuroblastoma Program website.


Seizures cause memory and cognitive loss by blocking expression of a neuronal calcium sensor (Oct 30, 2017)
Dr. Jeannie Chin, assistant professor at Baylor College of Medicine has identified a novel mechanism that shows how recurrent seizures impair memory and cognition.


Dr. Hsiao-Tuan Chao named the 2017 STAT Wunderkind (Oct 16, 2017)
Dr. Hsiao-Tuan Chao, an instructor of pediatrics - neurology at Baylor College of Medicine and a postdoctoral researcher at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, has been named a 2017 STAT Wunderkind.


Dr. Zoghbi receives The George W. Jacoby Lectureship (Oct 9, 2017)
The American Neurological Association has awarded The George W. Jacoby Lectureship to  Dr. Huda Zoghbi, professor in the departments of pediatrics, molecular and human genetics, neurology, and neuroscience at Baylor and director of the Jan and Dan DuncanNeurological Research Insitute at Texas Children'sHospital


Breakdown of brain cells’ metabolic collaboration linked to Alzheimer’s disease (Oct 1, 2017)
Researchers have discovered that impairing a critical partnership between brain cells can lead to neurodegeneration. 


Novel regulatory DNA loops found in gliomas: a promising target for future therapies (Sept, 19.2017)
Researchers in the laboratory of Dr. Benjamin Deneen, investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and associate professor at Baylor College of Medicine, have identified mechanisms in which NFIA, a glial gene, is regulated during normal brain development and in brain tumors. 


New study reveals how repeated early-life seizures alter microanatomy of neurons (Aug 24, 2017)
A recent study published in eNeuro from the laboratory of Dr. John Swann, director of the Gordon and Mary Cain Pediatric Neurology Research Foundation Laboratories at Texas Children’s Hospital shows how frequent seizures alter the microanatomy of neurons in the brain and that the activity of a specific enzyme may contribute to the cognitive and behavioral deficits observed in children with epileptic encephalopathies.


Next-generation probiotics may reduce the risk of gut bacterial infections (Aug 9, 2017)
C. difficile infections are the most common cause of diarrhea associated with the use of antibiotics and very difficult to eliminate. Texas Children's Drs. Jennifer Spinler and Tor Savidge in collaboration with co-author Dr. Robert A. Britton, professor of molecular virology and microbiology at Baylor and member of the Dan L Duncan Comprehensive Cancer Center have now found a way to selectively kill this bacteria using probiotics.


Ready-to-use cocktail of immune cells targeting specific viruses are safe and effective in transplant patients (Aug 8, 2017)
Transplant patients are at risk for developing potentially lethal viral infections, many of which are not easy to treat and carry significant side effects.  Researchers at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist have developed an alternative treatment in which virus-specific cells protect patients against severe, drug-resistant viral infections


Dr. Jimmy Holder awarded by the Doris Duke Charitable Foundation (July 26, 2017)
Dr. Jimmy Holder, a clinician and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and assistant professor at the Baylor College of Medicine, has been awarded the 2017 Clinical Scientist Development Award by the Doris Duke Charitable Foundation


Lunatic Fringe plays a key role in renewing neurons (July 26, 2017)
In a study published in the journal Elife, Dr. Mirjana Maletic-Savatic and colleagues describe how Lunatic Fringe, a Notch ligand helps to maintain the optimal number of neurons in the brain.


Patient-inspired research links sugar metabolism to a rare genetic disorder (June 29, 2017)
A team of researchers at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital have found Zellweger syndrome or peroxisomal biogenesis disorder is linked to altered sugar metabolism and not fat metabolism, as believed earlier.


Dr. Benjamin Arenkiel receives the 2017 Michael E. DeBakey, M.D 'Excellence in Research' Award (June 6, 2017)
Dr. Benjamin Arenkiel, a researcher at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s has received a 2017 Michael E. DeBakey, M.D., Excellence in Research Award, a prestigious honor given annually to Baylor College of Medicine faculty who have made the most significant published scientific contribution to clinical or basic biomedical research during the past three years.


Dr. Jordan Orange receives E. Mead Johnson Award for Research in Pediatrics (May 30, 2017)

Dr. Jordan Orange, MD, PhD, Professor in the Department of Pediatrics at Baylor College of Medicine and Texas Children's Hospital, where he serves as the Director of the Center for Human Immunobiology and the Vice Chair for Research, received the prestigious E. Mead Johnson Award for Research in Pediatrics from the American Pediatric Society and the Society for Pediatric Research.


Bellen Lab researcher receives the Harold M. Weintraub Graduate Student Award (May 24, 2017)

Dr. Lucy Liu, graduate student in Dr. Hugo Bellen's lab received the prestigious award for her work that showed how lipid metabolism causes neurodegeneration.


Dr. Sundeep Keswani receives the March of Dimes award (May 17, 2017)

Dr. Sundeep Keswani, surgical director of Basic Research and pediatric surgeon at Texas Children’s Hospital and associate professor of surgery in the Division of Pediatric Surgery at Baylor College of Medicine, was recently awarded a $300,000 grant over a period of three years for his project “Targeting the Extracellular Matrix: an Innovative Strategy to Improve Pulmonary Hypertension in Congenital Diaphragmatic Hernia


Researchers at the Jan and Dan Duncan Neurological Research Institute create a new web tool to expediate gene discovery (May 15, 2017)
A group of researchers led by Drs. Hugo Bellen and Zhandong Liu have created a newopen-access, user-friendly web resource, MARRVEL (Model Organism Aggregated Resource for Rare Variant ExpLoration) that will help biomedical researchers speed up their pace of scientific discovery. 


TCH/NRI physician-scientists receive prestigious research scholarships from the American Academy of Neurology (May 03, 2017)

Dr. Hsiao-Tuan Chao, MD PhD, medical resident of pediatric neurology and Dr. Laurie Robak, clinical instructor at the Baylor College of Medicine and researchers at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children's Hospital were selected to receive prestigious scholarship grants from the American Academy of Neurology to support studies on neurodevelopmental and neurodegenerative diseases


Research paves way for improved colorectal cancer test (April 19, 2017) 
The type of bacteria in your gut may help diagnose colorectal cancer. Researchers at Baylor College of Medicine and other institutions have identified specific types of bacteria that seem to be abundant in individuals with colorectal cancer. 


Texas Children's to offer brand new FDA-approved treatment for Spinal Muscular Atrophy (April 11, 2017)
Texas Children’s Hospital is among the first hospitals in the nation to offer the new FDA-approved treatment for Spinal Muscular Atrophy (SMA) to pediatric patients. Texas Children’s inaugurated this program in February 2017 for established and newly diagnosed SMA patients.


Mutations in an early embryonic development gene may cause female infertility (Mar 27, 2017)
A team of researchers headed by Dr. Ignatia Van den Veyver, professor and at Baylor College of Medicine, director of clinical prenatal genetics and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, have shown that mutations in an early embryonic development gene may provide some clue to unexplained female infertility and recurrent pregnancy loss. 


Mutations in ABL1, a known leukemia gene, could cause a novel developmental disorder (Mar 20, 2017)
Researchers at the Neurological Research Institute at Texas Children’s Hospital and faculty at Baylor College of Medicine have identified a novel role for ABL1, a known tumorigenic gene, in a new developmental disorder.


Rats could a valuable addition to an autism researcher's toolkit (Mar 15, 2017)
Mice are the mainstay of autism research. However, some researchers, including Drs. Rodney Samaco and Surabi Veeraraghavan of the Jan and Dan Duncan Neurological Research Institute and Baylor College of Medicine, argue in a lay article that rats are not merely "larger mice". 


Careful study of mouse models by Dr. Zoghbi's team has uncovered a new neurodevelopmental disorder in five patients (Mar 13, 2017)
A collaborative study spearheaded by Dr. Huda Zoghbi, professor at Baylor College of Medicine and director of Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, shows that loss of Ataxin1-Capicua (ATXN1-CIC) complex causes a previously unidentified neurodevelopmental disorder in mice and humans.


Texas Children's Hospital researchers identify five new candidate genes for Parkinson disease (Feb 20, 2017)
A large collaborative study led by Dr. Joshua Shulman, Assistant Professor at Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and Dr. Peter Heutink, professor at the German Center for Neurodegenerative Diseases, has revealed five strong candidate genes for Parkinson’s disease.


A potential therapeutic target for juvenile Batten disease (Feb 6, 2017)
Researchers in the laboratory of Dr. Marco Sardiello, assistant professor at Baylor College of Medicine and an investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital have identified a pharmacologically actionable target for juvenile Batten disease, a rare but fatal pediatric neurodegenerative disorder. 


Maternal diet alters energy and behavior of her adult male mice offspring (Jan 19, 2017)
A team of researchers in the laboratory of Dr. Ignatia Van den Veyver, professor at the Baylor College of Medicine and investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital found that adult male offspring of female mice that were fed chronic low-protein diet are sluggish, with increased body fat and anxiety-like behaviors even though they were on a normal diet since weaning. 


Fighting sickle cell disease with Type 2 diabetes medication (Jan 10, 2017)
Sickle cell disease and the blood disorder beta thalassemia affect more than 180,000 Americans and millions more worldwide. Both diseases can be made milder or even cured by increasing fetal hemoglobin (HbF) levels, but current treatment to ramp up HbF is limited in its effectiveness. 


Method of birth does not affect an infant's microbiome composition (Jan 23, 2017)
A new study published today by researchers at Baylor College of Medicine in Nature Medicine found that the microbiome of infants greatly expands between birth and 4 to 6 weeks of age. 


Researchers find that inhibitory neural networks broaden with age and experience (Jan 9, 2017)
For the first time, in a study published in Nature Neuroscience, researchers in the laboratory of Dr. Benjamin Arenkiel, associate professor at Baylor College of Medicine and principal investigator at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital, have demonstrated how inhibitory circuits form and develop in live brains. To learn more watch this video


Dr. Huda Zoghbi wins the prestigious Breakthrough Prize in Life Sciences (Dec 4, 2017)
Huda Zoghbi never dreamed her research into the rarest of rare brain disorders might one day lay the groundwork for promising therapeutic candidates for Alzheimer's and autism. She just wanted to help the patients she first encountered as a pediatrician-in-training in the 1980s, girls who mysteriously lost the ability to walk, speak and use their hands. 


Study reveals potential new strategy to prevent Alzheimer's disease (Oct 6, 2016)
Taking a pill that prevents the accumulation of toxic molecules in the brain might someday help prevent or delay Alzheimer’s disease, according to scientists at Baylor College of Medicine, Texas Children’s Hospital and Johns Hopkins University School of Medicine.


A small region of the brain plays a major role in controlling appetite (See the Wall Street Journal article, Oct 3, 2016).
A seminal study published in Nature from the laboratory of Dr. Benjamin Arenkiel, associate professor at Baylor College of Medicine and researcher at the Neurological Research Institute at Texas Children's Hospital, demonstrates that a subset of cholinergic neurons in the basal forebrain act as critical regulators to modulate food intake and control body weight.