ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026
Transient prophylactic immunosuppression with abatacept or dasatinib prevents immune responses in AAV gene transfer.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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1 citing paper in PubMed.
- Mechanisms of and mitigating strategies for cellular immune responses to CRISPR-associated nucleases in genome editing therapy.Frontiers in medicine · 2026Review
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15 authors.
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Abstract
Immune responses against the adeno-associated virus (AAV) vector capsid and transgene product pose significant hurdles to the efficacy and safety of gene therapy. Antibodies to the capsid can cause complement activation and prevent redosing, while T cell responses can mediate liver toxicity. Furthermore, responses against the transgene product can negatively impact therapy and cause tissue toxicities. Here, we investigated transient prophylactic immunosuppression at the time of AAV dosing as a means of mitigating immune responses, employing two clinically approved but mechanistically distinct drugs: abatacept, a CTLA-4-Ig fusion blocking T cell costimulation, and dasatinib, a small-molecule SRC kinase inhibitor that targets T cell receptor signaling. Results in mouse models demonstrate that both drugs can fully block neutralizing antibody formation against capsid, enable redosing by the systemic route, and suppress CD8
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