
Episode 384
Gene Therapy to Cure Rare Disease - Dr. Gaurav Shah
Talking Biotech with Dr. Kevin Folta · Colabra
February 18, 202335m 2s
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Show Notes
There are many rare diseases that originate with a single letter change in DNA, and they can cause tremendous physical and financial hardship for families affected. Viral-based gene therapies have been a promise for decades, and modern technology is now bringing these concepts to fruition. Dr. Gaurav Shah, CEO of Rocket Pharma, describes the power of the current gene therapy approaches, along with the pipeline of potential therapeutics that could represent cures for rare disease.
Topics
gene therapyviral vectorsAAVlentivirusorphan disease