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

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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Detalles Bibliográficos
Main Authors: Emily E. O’Driscoll, Sakshi Arora, Jonathan F. Lang, Beverly L. Davidson, Ophir Shalem
Formato: Artigo
Idioma:English
Publicado: Elsevier 2025-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
rAAV2
gene therapy
rAAV production
CRISPR screens
Acceso en liña:http://www.sciencedirect.com/science/article/pii/S2329050125000038

Internet

http://www.sciencedirect.com/science/article/pii/S2329050125000038

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