Efficient and sustained

CRISPR FOXP3 HSC editing IPEX gene editing hematopoietic stem cell

Journal

Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857

Informations de publication

Date de publication:
14 Mar 2024
Historique:
received: 14 06 2022
accepted: 20 12 2023
medline: 29 1 2024
pubmed: 29 1 2024
entrez: 29 1 2024
Statut: epublish

Résumé

Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a monogenic disorder caused by mutations in the

Identifiants

DOI: 10.1016/j.omtm.2023.101183 PMID: 38282895 PMC: PMC10818254
pubmed: 38282895
doi: 10.1016/j.omtm.2023.101183
pii: S2329-0501(23)00222-X
pmc: PMC10818254
doi:

Types de publication

Journal Article

Langues

eng

Pagination

101183

Informations de copyright

© 2023 The Author(s).

Déclaration de conflit d'intérêts

S.S., I.F.K, Y.H., K.M.S., and D.J.R hold provisional patent PCT WO2019210042 entitled “Expression of FOXP3 in edited CD34+ cells” related to this work. Another patent, “Expression of Human FOXP3 in Gene Edited T Cells,” has also been filed with Y.H., K.M.S., I.F.K, and D.J.R. as authors.

Auteurs

Swati Singh (S)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Cole M Pugliano (CM)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Yuchi Honaker (Y)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Aidan Laird (A)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

M Quinn DeGottardi (MQ)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Ezra Lopez (E)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Stefan Lachkar (S)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Claire Stoffers (C)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Karen Sommer (K)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

Iram F Khan (IF)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.

David J Rawlings (DJ)

Center for Immunity and Immunotherapies and the Program for Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA 98101, USA.
Department of Pediatrics, University of Washington, Seattle, WA 98101, USA.
Department of Immunology, University of Washington, Seattle, WA 98101, USA.

Classifications MeSH