Gene therapy has experienced an increasing number of successful human clinical trials, leading to 6 FDA approved products using delivery vectors based on adeno-associated viruses (AAV). These ...
Gene therapy has experienced an increasing number of successful human clinical trials, leading to 6 FDA approved products using delivery vectors based on adeno-associated viruses (AAV). These ...
EMERYVILLE, Calif., May 04, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene ...
The low efficiency of conventional vectors used for genetic medicines often means delivering them at high doses and by suboptimal routes, leading to inflammation, toxic side effects and limited ...
CNS-directed AAV gene therapy shows milder immune responses compared to systemic administration, reducing peripheral immune activation and systemic toxicities. Direct CNS delivery can bypass ...
The 2012 landmark Science paper demonstrating the use of CRISPR-Cas9 as a gene-editing tool catapulted gene-editing technologies to new levels. Yet, that breakthrough also highlighted precision and ...
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