Scientific Advisory Board

SCIENTIFIC ADVISORY BOARD

Dr. Wendy Chung

Wendy Chung, M.D., Ph.D. is a clinical and molecular geneticist and the Kennedy Family Professor of Pediatrics and Medicine at Columbia University. She received her B.A. in biochemistry and economics from Cornell University, her M.D. from Cornell University Medical College, and her Ph.D. from The Rockefeller University in genetics. She described Okur-Chung syndrome in 2016 with Dr. Okur. Dr. Chung directs NIH funded research programs in human genetics of autism, neurodevelopmental disorders, birth defects including congenital diaphragmatic hernia and congenital heart disease, obesity, breast cancer, and pulmonary hypertension. She leads the Precision Medicine Resource in the Irving Institute At Columbia University.


She has authored over 300 peer reviewed papers and 50 reviews and chapters in medical texts. She was the recipient of the American Academy of Pediatrics Young Investigator Award, the Medical Achievement Award from Bonei Olam, and a career development award from Doris Duke. Dr. Chung is renowned for her teaching and mentoring and received Columbia University’s highest teaching award, the Presidential Award for Outstanding Teaching. She was the original plaintiff in the Supreme Court case that overturned the ability to patent genes and served on the Institute of Medicine Committee on Genetic Testing.


Dr. Chung enjoys the challenges of genetics as a rapidly changing field of medicine and strives to facilitate the integration of genetic medicine into all areas of health care in a medically, scientifically, and ethically sound, accessible, and cost effective manner.

Dr. Volkan Okur

Dr. Volkan Okur completed his medical school education and medical genetics residency training in Turkey. He has experience in clinical, diagnostic, and research fields of human genetics. He has been working at Columbia University since June 2015 with Dr. Wendy Chung in discovering underlying genetic causes of neurodevelopmental diseases. Among his interests are dysmorphology, genetic counseling, next-generation sequencing, single-gene diseases, chromosomal diseases, population genetics, and rare disease advocacy.

Kyle Retterer

Kyle Retterer is the Vice President of Genomic Science at Geisinger. He holds a B.S. in Physics and M.S. in Biotechnology and Bioinformatics from the University of Maryland.


Kyle has authored over 30 peer-reviewed articles primarily focused on whole exome sequencing, bioinformatics, and the discovery of novel disease genes. His areas of expertise include assay design, pipeline development, genomic analysis automation, and machine learning. Kyle’s research interests include neurodevelopmental disorders, aortic dissection, and the application of deep learning to clinical genomics.

Etty (Tika) Benveniste, Ph.D.

Etty (Tika) Benveniste, Ph.D. is Senior Vice Dean for Research, UAB Heersink School of Medicine. She was the Founding Chair of the Department of Cell, Developmental and Integrative Biology (2012-2015), and served as Chair of the Department of Cell Biology (2000-2011). Dr. Benveniste was the Founding Associate Dean for Postdoctoral Education (1999-2001) and Director, Graduate Program in Cell Biology (1995-2000).


Dr. Benveniste initiated research as a postdoctoral fellow at UCLA in the emerging field of neuroimmunology, elucidating mechanisms by which the immune system and the central nervous system communicate and influence functionality. Specifically, her lab has focused on the interaction of T-cells, neutrophils, and macrophages with cells of the central nervous system (microglia, astrocytes). Specifically, her work focuses on signaling cascades mediated by the JAK/STAT pathway and Protein Kinase CK2 in terms of altering the functionality of immune cells and glial cells. These studies have implications for autoimmune/neurodegenerative diseases such as Multiple Sclerosis and Parkinson’s Disease. Her laboratory has over 250 publications on these topics in highly-ranked journals. Dr. Benveniste’s research is supported by grants from the NIH and the Michael J. Fox Foundation. Dr. Benveniste was elected in 2009 as a Fellow of the American Association for the Advancement of Science (AAAS).


Dr. Benveniste takes great pride in her mentorship activities, which include the training/mentoring of over 270 high school students, undergraduates, graduate students, medical students, residents, postdoctoral fellows and junior faculty. Thirty-six graduate students have obtained their Ph.D. degree from Dr. Benveniste’s lab, with 1 currently training and 34 postdoctoral fellows have trained with her, with 2 currently training. In 2014, Dr. Benveniste received the UAB Graduate School Dean’s Award for Excellence in Mentoring.

Dr. Rachel Bailey

Dr. Rachel Bailey received dual Bachelor of Science degrees in Biology/Bioinformatics and Molecular Biology from Rensselaer Polytechnic Institute. She began her Ph.D. in Neuroscience at the Mayo Clinic and completed it at the University of Florida under the mentorship of Dr. Jada Lewis. Dr. Bailey’s graduate work focused on studying modifiers of tauopathies. She demonstrated that the LRRK2 protein that is linked to Parkinson’s disease directly phosphorylates tau protein at a novel epitope, which is an important step in the neurodegenerative process.

 

For her postdoctoral fellowship, Dr. Bailey worked in the Gene Therapy Center at the University of North Carolina Chapel Hill in the lab of Dr. Steven Gray. She contributed to the preclinical development of an AAV-based gene therapy for the rare pediatric neurodegenerative disorder, Giant Axonal Neuropathy (GAN), which is currently being tested in a Phase I clinical trial that is ongoing at the Clinical Center at the NIH (NCT02362438). Dr. Bailey also characterized autonomic nervous system dysfunction in GAN rodent models and optimized targeting AAV vector to the peripheral nervous system in these models. 

 

Now at UT Southwestern, the research in the Bailey lab focuses on the development of gene therapies for neurological disorders and facilitates the translation of these treatments for use in human. Pediatric disorders that she is working on include SLC13A5 epileptic encephalopathy, Multiple Sulfatase Deficiency, Charcot Marie Tooth disease type 4J and GAN. Dr. Bailey has expanded her gene therapy research efforts beyond monogenic diseases to more complex neurodegenerative disorders, including tauopathies, such as Alzheimer’s disease. Her lab utilizes AAV vector engineering for both gene replacement and gene-silencing approaches, explores alternative methods of virus delivery, performs proof-of-concept studies in cell culture models, and performs IND-enabling studies in animal models for translation of treatments to the clinic.

Dr. Goodspeed

Dr. Goodspeed is a Child Neurologist and Neurodevelopmental Specialist who cares for patients and families with autism spectrum disorder and intellectual disability. Her research focuses on defining the natural history rare genetic neurodevelopmental disorders and developing disease-related outcome measures and biomarkers to prepare for future clinical trials.  She also has expertise in clinical trial development for genetic therapies and aims to bring targeted therapies to patients who have very few options.

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