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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...

Ausführliche Beschreibung

Gespeichert in:
Hauptverfasser: Emily E. O’Driscoll, Sakshi Arora, Jonathan F. Lang, Beverly L. Davidson, Ophir Shalem
Format: Artigo
Sprache:Inglês
Veröffentlicht: Elsevier 2025-03-01
Schriftenreihe:Molecular Therapy: Methods & Clinical Development
Schlagworte:
AAV
rAAV2
gene therapy
rAAV production
CRISPR screens
Online-Zugang:http://www.sciencedirect.com/science/article/pii/S2329050125000038
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