Article

Split intein-mediated protein trans-splicing to express large dystrophins

2023-06-01

Abstract excerpt

<title>Abstract</title> <p>Gene replacement using Adeno-associated viral (AAV) vectors represents a promising approach for many diseases. However, this therapeutic modality is challenged by the modest packaging capacity of AAVs (~4.7 kb), limiting its application for disorders involving large genes, such as Duchenne muscular dystrophy with a coding sequence >11 kb. Here we developed a novel method for expressing...

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Literature Corpus work: aac7f744-b4ed-5577-b182-e39efc703a91
DOI: 10.21203/rs.3.rs-2924001/v1

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