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Penn Researchers Develop Approach to Prevent Toxicity Tied to Neurological Gene Therapy

Penn Medicine researchers have developed a new targeted approach to prevent a toxicity seen in the sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. It’s an important hurdle to clear, as the field works toward more safe and effective gene therapies for patients with disorders like spinal muscular atrophy.


Philadelphia is poised to become the epicenter of cell and gene therapy

THE 7,600-JOB MOMENT Philadelphia is poised to become the epicenter of cell and gene therapy. Leaders like Dr. Steven Nichtberger have brought the local industry to a ‘tipping point.’


A quest for Quincy: Gene therapies come of age for some forms of autism

Allyson Berent is a specialty veterinarian in New York City. She treats animals that other doctors cannot help. When no good therapies are available, she invents one. Cats and dogs consumed almost all of her time — until six years ago, when her second daughter was born.


The Wilderness of Rare Genetic Diseases and the Parents Navigating It

“Rare parents” are often left charting a world of unknowns. But their persistence has paid off with more advanced treatment options.


Harrington Discovery Institute Announces Award Recipients in its COVID-19 Rapid Response Initiative

Dr. Jim Wilson has been selected by The Harrington Discovery Institute for a research award in the COVID-19 Rapid Response Initiative.


Gene Therapy Program at Penn Medicine Joins AAVCOVID Vaccine Project

Renowned program led by James M. Wilson, MD, PhD, a leading expert in the technology platform used in the experimental vaccine, to conduct preclinical studies in joint research and development project with Massachusetts Eye and Ear and Massachusetts General Hospital.