Targeted knock-in of

CRISPR-Cas9 HDR-mediated knock-in MT: RNA/DNA Editing NCF1 NCF2 adeno-associated viral vectors chronic granulomatous disease gene editing gene therapy hematopoietic stem cells integration-deficient lentiviral vectors

Journal

Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621

Informations de publication

Date de publication:
10 Sep 2024
Historique:
received: 03 10 2023
accepted: 22 05 2024
medline: 2 7 2024
pubmed: 2 7 2024
entrez: 2 7 2024
Statut: epublish

Résumé

p47

Identifiants

DOI: 10.1016/j.omtn.2024.102229 PMID: 38952440 PMC: PMC11215332
pubmed: 38952440
doi: 10.1016/j.omtn.2024.102229
pii: S2162-2531(24)00116-1
pmc: PMC11215332
doi:

Types de publication

Journal Article

Langues

eng

Pagination

102229

Informations de copyright

© 2024 The Authors.

Déclaration de conflit d'intérêts

The authors declare no competing interests.

Auteurs

Kah Mun Siow (KM)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Merve Güngör (M)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Dominik Wrona (D)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Federica Raimondi (F)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Oleksandr Pastukhov (O)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Panagiotis Tsapogas (P)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Timon Menzi (T)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Michael Schmitz (M)

Department of Biochemistry, University of Zurich, 8057 Zurich, Switzerland.

Péter István Kulcsár (PI)

Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland.

Gerald Schwank (G)

Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland.

Ansgar Schulz (A)

Department of Pediatrics, University Medical Center Ulm, 89075 Ulm, Germany.

Martin Jinek (M)

Department of Biochemistry, University of Zurich, 8057 Zurich, Switzerland.

Ute Modlich (U)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.

Ulrich Siler (U)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.
School of Life Sciences, Institute for Pharma Technology, University of Applied Sciences and Arts Northwestern Switzerland, 4132 Muttenz, Switzerland.

Janine Reichenbach (J)

Division of Gene and Cell Therapy, Institute for Regenerative Medicine, University of Zurich, Schlieren, 8952 Zurich, Switzerland.
Department of Somatic Gene Therapy, University Children's Hospital Zurich, 8032 Zurich, Switzerland.
Center for Applied Biotechnology and Molecular Medicine (CABMM), University of Zurich, 8057 Zurich, Switzerland.

Classifications MeSH