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Updated: Sep 8, 2025

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Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification
Published on: October 11, 2024
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The Responsible Development of Adeno-Associated Virus Gene Therapies
James M Wilson1, Arthur L Caplan2
1GEMMA Biotherapeutics Inc., Philadelphia, Pennsylvania, USA.
Human Gene Therapy
|July 11, 2025
Abstract:
Several unexpected fatalities in patients who received adeno-associated virus (AAV)-based gene therapies have recently occurred. These tragic events have cast a pall over the entire sector with some stakeholders suggesting that AAV is patently unsafe as a gene delivery platform and ought not to be pursued. This conclusion is not warranted.

