Application of a Spacer-nick Gene-targeting Approach to Repair Disease-causing Mutations with Increased Safety.

Tran, Ngoc Tung; Lebedin, Mikhail; Danner, Eric; Kühn, Ralf; Rajewsky, Klaus; Chu, Van Trung

Tran, Ngoc Tung; Lebedin, Mikhail; Danner, Eric; Kühn, Ralf; Rajewsky, Klaus; Chu, Van Trung.

Afiliação

Tran NT; Immune Regulation and Cancer, Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.
Lebedin M; Immune Mechanisms and Human Antibodies, Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.
Danner E; Genome Engineering & Disease Models, Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.
Kühn R; Genome Engineering & Disease Models, Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.
Rajewsky K; Immune Regulation and Cancer, Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.
Chu VT; Immune Regulation and Cancer, Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.

Bio Protoc ; 13(8): e4661, 2023 Apr 20.

Article em En | MEDLINE | ID: mdl-37113334

Palavras-chave

AAV6; Cas9D10A nickase; Gene correction efficiency; Gene repair approach; Human hematopoietic stem and progenitor cells (HSPCs); Off-target mutations; PAM-out sgRNAs; Spacer distance of 200-350 bp; Unintended on-target mutations

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Guideline Idioma: En Revista: Bio Protoc Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Alemanha País de publicação: Estados Unidos

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