Article

Characterization of Immune Responses to rAAVrh8 Gene Therapy for GM2 Gangliosidosis in Phase 1/2 Trial

2025-05-29

Abstract excerpt

<title>Abstract</title> <p>Understanding how the immune system responds to adreno-associated virus (AAV) gene therapy and potentially modulating that response is vital to their safety and ultimate success. However, the immune response in the central nervous system (CNS) to AAV gene therapy is still not well understood. Here, we characterized the immune responses to AAVrh8 vectors injected into the thalamus and ce...

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Literature Corpus work: 65f0aecb-2970-54c0-a5af-7ecdde27f6d3
DOI: 10.21203/rs.3.rs-6431041/v1

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