
Muscular dystrophy gene therapy nears approval, but safety concerns linger Use of viruses poses significant risks and, for now, prevents retreatment if benefits fade
J Kaiser, Science, May 23, 2023
Emerging Trends in Gene Therapy: Thalassemia as a Case Study
CDC Seminar— June 22, 2023, 2:00–3:00 PM ET
The gene-therapy revolution risks stalling if we don't talk about drug pricing.
et al. Nature 2023 4 (7958) 629-630
Contributions from medical geneticists in clinical trials of genetic therapies: A points to consider statement of the American College of Medical Genetics and Genomics (ACMG)
LDM Pena et al, Genetics in Medicine, April 9, 2023
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