Article

A self-inactivating system for AAV-mediated in vivo base editing

2022-05-20

Abstract excerpt

<title>Abstract</title> <p>DNA base editors have been harnessed as an exciting therapeutic platform for human diseases and are rapidly progressing into human clinical trials. However, persistent expression of base editors delivered via adeno-associated virus (AAV) poses concerns with specificity and immunogenicity. Here we develop self-inactivating base editor (siBE) systems with a negative feedback loop where on...

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Literature Corpus work: 7a9b34b6-4a98-507f-acfe-9f1a69226aca
DOI: 10.21203/rs.3.rs-1663604/v1

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