Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteintrans-splicing in the retina

Split intein-mediated protein trans-splicing expands AAV transfer capacity, thus overcoming the limited AAV cargo. However, non-mammalian inteins persist as trans-splicing by-products, and this could raise safety concerns for AAV intein clinical applications. In this study, we tested the ability of...

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Auteurs principaux:	Patrizia Tornabene, Ivana Trapani, Miriam Centrulo, Elena Marrocco, Renato Minopoli, Mariangela Lupo, Carolina Iodice, Carlo Gesualdo, Francesca Simonelli, Enrico M. Surace, Alberto Auricchio
Format:	Artigo
Langue:	Inglês
Publié:	Elsevier 2021-12-01
Collection:	Molecular Therapy: Methods & Clinical Development
Sujets:	protein trans-splicing split-inteins intein degradation ecDHFR gene therapy AAV
Accès en ligne:	http://www.sciencedirect.com/science/article/pii/S2329050121001613
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