Leadership – ENDD

8Aug

Beverly Davidson, PhD

Dr Davidson is the Director of Translational Research at ENDD, the Chief Scientific Strategy Officer and Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and the Arthur V. Meigs endowed Chair in Pediatrics at the Children’s Hospital of Philadelphia. She is also Professor of Pathology and Laboratory Medicine and Professor of Genetics at the Perelman School of Medicine, University of Pennsylvania.

In 2019, Dr Davidson was elected a member of the National Academy of Medicine for her “being on the forefront of developing innovative therapies and medicines for fatal, inherited brain disorders, which are engineered to either remove toxic proteins or replace missing proteins, and for improving or preventing disease progression.”

Dr Davidson received her B.S. in Biology from Nebraska Wesleyan University, and Ph.D. in Biological Chemistry from University of Michigan. She completed postdoctoral studies in Molecular Genetics at the University of Michigan. Recent honors include election into the American Academy of Arts. She is the current president of the American Society of Gene and Cell Therapy, the largest international association of gene and cell therapy research.

Lab Members

Defne Amado, MD, PhD

Assistant Professor of Neurology

Sakshi Arora, PhD

Research Associate

Brandon Brown, PhD

Postdoctoral Fellow

Guillem Chillón, BS, BA

Graduate Student

Max Gessner, BS

Research Technician

Icnelia Huerta, MD, PhD

Lab Manager

Shareen Nelson, BS

Research Assistant

8Aug

Deborah French, PhD

Dr. French manages the human stem cell model development of ENDD. She is also Director of the Human Pluripotent Stem Cell Core at The Children’s Hospital of Philadelphia (CHOP) and a Research Professor of Pathology and Laboratory Medicine at the Perelman School of Medicine, University of Pennsylvania.

Dr. French has extensive experience in the field of pluripotent stem cell biology and established the CHOP core in 2008. She has research experience in the areas of Immunology, Hematology, and Cell Biology with areas of expertise including megakaryocytes and platelets, integrin adhesion receptors, and pluripotent stem cell biology. Her primary research focus is in the field of platelet biology, though as Director of the Human Pluripotent Stem Cell Core she has been centrally involved in a host of significant scientific collaborations. She has built a solid foundation for modeling human disease to study mechanism, development, and establish new therapeutic modalities.

At ENDD, Dr. French oversees human model development including induced pluripotent stem cell generation, characterization, and quality control; differentiation to the appropriate germ layer and derivative lineage, including hematopoietic and neuronal; and gene editing using the CRISPR/Cas9 technology. The ability to edit genes using the CRISPR/Cas9 technology has been a game-changer in the field and is being utilized on established pluripotent stem cell lines for creating isogenic control and variant pairs or an allelic series of gene variants on a uniform genetic background for genotype-phenotype studies.

Lab Members

Lea Dungan, BS

Dulcie Lai, PhD, PharmD

Giulia Pavani, PhD

Elisa Waxman, PhD

8Aug

Elizabeth Heller, PhD

The Heller Lab research program is focused on elucidating the causal mechanisms by which epigenetic reprogramming contributes to neuropsychiatric and neurodevelopmental disease. The Heller lab applies targeted epigenetic editing to determine the direct causal relevance of histone modifications to transcription, alternative splicing, and behavior. This approach will be applied to SynGAP1, to define gene regulatory mechanisms and contribute to novel therapeutic approaches. Dr. Heller is an Associate Professor of Pharmacology at the University of Pennsylvania Perelman School of Medicine. She received her PhD from The Rockefeller University and completed her postdoctoral fellowship at the Icahn School of Medicine at Mount Sinai. Dr. Heller is the recipient of a 2017 NIH Director’s Pioneer Award, a 2020 SynGAP Research Fund Grant, and a 2023 Orphan Disease Center Million Dollar Bike Ride Award from the Syngap Research Fund.

Lab Members

Molly Reilly, PhD

Postdoctoral Fellow

Laura Sichlinger, PhD

Postdoctoral Fellow

8Aug

Ingo Helbig, MD

Dr Helbig is the Director of Clinical Research at ENDD, an attending Pediatric Neurologist in the Division of Neurology and the Director of Genomic Science at Children’s Hospital of Philadelphia (CHOP), and an Assistant Professor of Neurology at the Perelman School of Medicine, University of Pennsylvania.

Dr Helbig co-headed the EuroEPINOMICS-RES Consortium, the European counterpart of the NIH-funded Epi4K consortium involved in collaborative genomic studies to identify genes for human epilepsies. He was part of the Genetics Commission of the International League Against Epilepsy (ILAE) and the Epilepsiome Task Force of the ILAE Genetics Commission, which aims to increase genetic literacy in the epilepsy community. He is an expert in clinical genetics and clinical care for several monogenic neurodevelopmental disorders. His work has led to greater understanding of how genetic changes lead to severe epilepsies including STXBP1, SYNGAP1, GRIN2A, CHD2, KCNA2, HCN1, and DNM1. Recently, his focus has shifted to development of clinical outcome measures and biomarkers for clinical trials.

He received an MD at Ruprechts-Karl University Heidelberg, Medical Faculty Mannheim, Mannheim, Germany. He completed a fellowship in Pediatric Epileptology at the Epilepsy Research Centre, Melbourne, Australia, and did residencies in Pediatrics and Neuropediatrics at the University Hospital Schleswig-Holstein, Germany, and Child Neurology at CHOP.