Proceedings of the National Academy of Sciences of the United States of America

About the PNAS Member Editor
Name Bennett, Jean
Location University of Pennsylvania, Perelman School of Medicine
Primary Field Medical Physiology and Metabolism
Secondary Field Cellular and Molecular Neuroscience
 Election Citation
Bennett studies the pathogenesis of inherited retinal degenerations and the potential applications of gene transfer to treat blinding disease.
 Research Interests
Jean Bennett's laboratory is interested in the pathogenesis of inherited retinal degenerations and the potential applications of gene transfer for ameliorating or even curing blinding disease. The laboratory has thus evaluated safety and durability of gene transfer to various retinal cell types, stages of development, and in different model systems, immune responses, different inherited and acquired forms of retinal disease, and approaches to treating blinding disease using a variety of gene-based approaches. Her team completed both a re-administration study, first in animals and then with Spark Therapeutics (of which she is a scientific founder), and a registrational gene therapy trial for congenital blindness, the first randomized, controlled, multi-center gene therapy Phase 3 gene therapy trial targeting a genetic disease. This work led to the first approved in vivo gene therapy for inherited disease in USA (2017), in Europe (2018), and now in multiple continents. Dr. Bennett established a "from bench to bedside" program using her experience in moving successful laboratory findings through rigorous safety and efficacy testing and, if warranted, into clinical trials. Dr. Bennett continues to develop gene-based therapies for retinal degenerative diseases and to tackle some of the limitations of current gene therapy technologies.

 
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