Although Wilson first became known to many after the death of a trial participant, his unwavering focus on curing rare diseases has fueled major progress in the field.
after 33 years for the private sector. He’s been named CEO of GEMMA Biotherapeutics, a biotech company, and board chairman of Franklin Biolabs, which provides diagnostic testing to gene therapy companies.Wilson’s time at Penn was dedicated to finding gene-based cures for rare diseases. He faced significant hurdles along the way but is now known as one of the preeminent experts in the field.
Jesse Gelsinger had a genetic deficiency in a crucial liver enzyme that, in its most serious form, can be fatal within a few days of birth. Gelsinger’s form was relatively mild, which enabled him to live a mostly normal life, provided heAs part of the trial, Gelsinger received a therapy consisting of a corrected version of the liver enzyme gene inside a modified virus that could deliver it into his cells.
What’s more, if a gene therapy cures a disease, patients only need to take it once, rather than a steady supply of pills over their lifetimes. Companies end up charging a lot for each dose to make the time and effort to develop a cure for a rare disease worthwhile.To protect future trial participants from what happened to Gelsinger, Wilson began using different viruses to deliver corrected genes into cells without triggering an immune reaction in patients.
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