Article

Transient costimulatory blockade overcomes immune barriers to durable and redosable liver-directed gene therapy

2026-07-27

Abstract excerpt

<title>Abstract</title> <p>In vivo liver-directed gene therapy (GT) is a therapeutic option for the permanent correction of monogenic diseases. Lentiviral vectors (LV), which integrate into the target cells' genome, represent a unique platform for intervention in childhood, with negligible limitations from pre-existing immunity to the parental virus. However, adaptive immunity to the encoded transgene and vector...

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Literature Corpus work: 15275778-11a3-528b-9aec-e9c6a3f8d0cc
DOI: 10.21203/rs.3.rs-10368898/v1

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