CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production

Recombinant adeno-associated virus (rAAV) vectors are an effective and well-established tool in the growing gene therapy field, with five U.S. Food and Drug Administration-approved AAV-mediated gene therapies already on the market and numerous more in clinical trials. However, manufacturing rAAV vec...

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Autors principals:	Emily E. O’Driscoll, Sakshi Arora, Jonathan F. Lang, Beverly L. Davidson, Ophir Shalem
Format:	Artigo
Idioma:	Inglês
Publicat:	Elsevier 2025-03-01
Col·lecció:	Molecular Therapy: Methods & Clinical Development
Matèries:	AAV rAAV2 gene therapy rAAV production CRISPR screens
Accés en línia:	http://www.sciencedirect.com/science/article/pii/S2329050125000038
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