Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

Abstract excerpt

Recombinant adenovirus-associated (rAAV) vectors due to their ease of construction, wide tissue tropism, and lack of pathogenicity remain at the forefront for long-term gene replacement therapy. In spite of very encouraging preclinical results, clinical trials were initially unsuccessful; expression of the rAAV vector-delivered therapeutic protein was transient. Loss of expression was linked to an expansion of...

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