Dr. Davut Pehlivan awarded a prestigious research grant by the Doris Duke Foundation
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Davut Pehlivan, MD, assistant professor at Baylor College of Medicine and instructor of Neurology at Texas Children’s Hospital, was among 21 physician-scientists across the nation who recently received funding support from the Doris Duke Foundation (DDF) for advancing the prevention, diagnosis, and treatment of diseases by strengthening and supporting the clinical research.
Through fellowships and multi-year grants, the Foundation will award a combined $7.92 million towards mentored research training and protected time for early-career physician-scientists to advance clinically significant groundbreaking research. This year’s grantees join a network of accomplished physicians who have gone on to become leaders in academia, medicine, public health, and government.
“Davut is an outstanding clinical investigator committed to advancing translational and clinical research and this grant will help him towards this goal. He is exactly the kind of highly-trained physician-scientist we need in medicine today – someone who can translate the many exciting basic discoveries to new and viable therapies that will help patients”. Dr. Huda Zoghbi, distinguished service professor at Baylor College of Medicine and founding director of the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, said.
A practicing child neurologist, Dr. Pehlivan specializes in diagnosing and treating neurogenetic and neurodevelopmental disorders in children from birth to young adulthood. As a clinician/scientist, he is particularly keen to focus on rare and undiagnosed genetic disorders, to discover precise diagnoses and to help develop new curative treatments. He was selected to receive the DDF grant for his project entitled, “Deep Phenotyping and Genomic Studies to Guide Gene-based Therapies in Dosage Sensitive Genomic Disorders.” The goals of this project are to
- Establish the path for personalized medicine in gene therapy-based treatments
- Understand the factors that cause differences in clinical severity in neurogenetic disorders
- Improve translational and clinical research in rare disorders by performing comprehensive clinical and cutting-edge genomic studies in MECP2- and RAI-1-related disorders.
Loss or excessive MECP2 results in distinct neurogenetic disorders. Thus, any treatment to restore MECP2 levels must be precise. By conducting deep and longitudinal assessments of the children enrolled in clinical studies, Dr. Pehlivan plans to develop clinical outcome measures, conduct natural history studies, and unravel subtle clinical differences in allelic disorders. The data obtained from these studies will uncover key insights for future clinical trials of gene (or other) therapies, such as identifying the biomarkers critical for delivering the exact dose of gene-based treatments for MECP2 and other disorders that are highly sensitive to gene dosage. Thus, the successful completion of this proposed project is expected to establish a translational/clinical research platform for rare neurogenetic disorders and carve a path toward personalized medicine.
“Dr. Pehlivan is one of the most capable young clinician-investigators I have known. His dedication to patients who suffer from rare genetic disorders has no bounds,” Dr. Mirjana Maletic-Savatic, professor at Baylor College and Duncan NRI investigator and a mentor. “As a neurogeneticist, he brings critical knowledge to every single patient he evaluates and has deep understanding of the fundamental processes that lead to their pathology. I am very happy that he was selected for this prestigious award and I have no doubts that his work will lead to transformational shifts in gene therapy for the patients he is studying.”
A complete list of this year’s DDF grant awardees can be found here.
About the Doris Duke Foundation
The Doris Duke Foundation (DDF) supports the well-being of people and the planet for a more creative, equitable, and sustainable future. We operate five national programs—in the performing arts, the environment, medical research, child and family well-being, and mutual understanding between communities—as well as Duke Farms and Shangri La, two centers that directly serve the public. Through the Medical Research Program, DDF strives to advance the prevention, diagnosis, and treatment of human disease by strengthening and supporting clinical research.
Davut Pehlivan, MD, assistant professor at Baylor College of Medicine and instructor of Neurology at Texas Children’s Hospital, was among 21 physician-scientists across the nation who recently received funding support from the Doris Duke Foundation (DDF) for advancing the prevention, diagnosis, and treatment of diseases by strengthening and supporting the clinical research.
Through fellowships and multi-year grants, the Foundation will award a combined $7.92 million towards mentored research training and protected time for early-career physician-scientists to advance clinically significant groundbreaking research. This year’s grantees join a network of accomplished physicians who have gone on to become leaders in academia, medicine, public health, and government.
“Davut is an outstanding clinical investigator committed to advancing translational and clinical research and this grant will help him towards this goal. He is exactly the kind of highly-trained physician-scientist we need in medicine today – someone who can translate the many exciting basic discoveries to new and viable therapies that will help patients”. Dr. Huda Zoghbi, distinguished service professor at Baylor College of Medicine and founding director of the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, said.
A practicing child neurologist, Dr. Pehlivan specializes in diagnosing and treating neurogenetic and neurodevelopmental disorders in children from birth to young adulthood. As a clinician/scientist, he is particularly keen to focus on rare and undiagnosed genetic disorders, to discover precise diagnoses and to help develop new curative treatments. He was selected to receive the DDF grant for his project entitled, “Deep Phenotyping and Genomic Studies to Guide Gene-based Therapies in Dosage Sensitive Genomic Disorders.” The goals of this project are to
- Establish the path for personalized medicine in gene therapy-based treatments
- Understand the factors that cause differences in clinical severity in neurogenetic disorders
- Improve translational and clinical research in rare disorders by performing comprehensive clinical and cutting-edge genomic studies in MECP2- and RAI-1-related disorders.
Loss or excessive MECP2 results in distinct neurogenetic disorders. Thus, any treatment to restore MECP2 levels must be precise. By conducting deep and longitudinal assessments of the children enrolled in clinical studies, Dr. Pehlivan plans to develop clinical outcome measures, conduct natural history studies, and unravel subtle clinical differences in allelic disorders. The data obtained from these studies will uncover key insights for future clinical trials of gene (or other) therapies, such as identifying the biomarkers critical for delivering the exact dose of gene-based treatments for MECP2 and other disorders that are highly sensitive to gene dosage. Thus, the successful completion of this proposed project is expected to establish a translational/clinical research platform for rare neurogenetic disorders and carve a path toward personalized medicine.
“Dr. Pehlivan is one of the most capable young clinician-investigators I have known. His dedication to patients who suffer from rare genetic disorders has no bounds,” Dr. Mirjana Maletic-Savatic, professor at Baylor College and Duncan NRI investigator and a mentor. “As a neurogeneticist, he brings critical knowledge to every single patient he evaluates and has deep understanding of the fundamental processes that lead to their pathology. I am very happy that he was selected for this prestigious award and I have no doubts that his work will lead to transformational shifts in gene therapy for the patients he is studying.”
A complete list of this year’s DDF grant awardees can be found here.
About the Doris Duke Foundation
The Doris Duke Foundation (DDF) supports the well-being of people and the planet for a more creative, equitable, and sustainable future. We operate five national programs—in the performing arts, the environment, medical research, child and family well-being, and mutual understanding between communities—as well as Duke Farms and Shangri La, two centers that directly serve the public. Through the Medical Research Program, DDF strives to advance the prevention, diagnosis, and treatment of human disease by strengthening and supporting clinical research.
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