Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
B lymphocytes
CD34+ cells
RAG1
SCID
T lymphocytes
gene rearrangement
gene therapy
lentiviral vector
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:
12 Jun 2020
12 Jun 2020
Historique:
received:
13
03
2020
accepted:
23
03
2020
entrez:
24
4
2020
pubmed:
24
4
2020
medline:
24
4
2020
Statut:
epublish
Résumé
Recombinase-activating gene-1 (RAG1)-deficient severe combined immunodeficiency (SCID) patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. Gene therapy is an alternative for those RAG1-SCID patients who lack a suitable bone marrow donor. We designed lentiviral vectors with different internal promoters driving codon-optimized
Identifiants
pubmed: 32322605
doi: 10.1016/j.omtm.2020.03.016
pii: S2329-0501(20)30046-2
pmc: PMC7163047
doi:
Types de publication
Journal Article
Langues
eng
Pagination
666-682Informations de copyright
© 2020 The Authors.
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