LYMPHOHEMATOPOIETIC ENGINEERING USING CAS9 BASE EDITORS

LYMPHOHEMATOPOIETIC ENGINEERING USING CAS9 BASE EDITORS

Provided herein are methods and systems for targeted gene disruption (knock-out, missense mutation) and targeted gene knock-in in mammalian cells using base editors and guide RNAs (gRNAs) designed to target splice acceptor-splice donor sites. Also provided herein are universally acceptable genetical...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Diers, Miechaleen, Lahr, Walker, Lonetree, Cara-Lin, Pomeroy, Emily Joy, Moriarity, Branden, Webber, Beau, Kluesner, Mitchell
Format: Patent
Sprache:eng
Schlagworte:
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Beschreibung
Zusammenfassung:Provided herein are methods and systems for targeted gene disruption (knock-out, missense mutation) and targeted gene knock-in in mammalian cells using base editors and guide RNAs (gRNAs) designed to target splice acceptor-splice donor sites. Also provided herein are universally acceptable genetically engineered cells comprising targeted disruptions in immunotherapy-related genes and comprising a CAR/TCR for therapeutic applications.