Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial.
Journal
Nature medicine
ISSN: 1546-170X
Titre abrégé: Nat Med
Pays: United States
ID NLM: 9502015
Informations de publication
Date de publication:
01 2022
01 2022
Historique:
received:
28
02
2021
accepted:
23
09
2021
pubmed:
5
1
2022
medline:
23
2
2022
entrez:
4
1
2022
Statut:
ppublish
Résumé
β-Thalassemias are inherited anemias that are caused by the absent or insufficient production of the β chain of hemoglobin. Here we report 6-8-year follow-up of four adult patients with transfusion-dependent β-thalassemia who were infused with autologous CD34
Identifiants
pubmed: 34980909
doi: 10.1038/s41591-021-01554-9
pii: 10.1038/s41591-021-01554-9
pmc: PMC9380046
mid: NIHMS1827715
doi:
Substances chimiques
Antigens, CD34
0
Globins
9004-22-2
Banques de données
ClinicalTrials.gov
['NCT01639690']
Types de publication
Clinical Trial, Phase I
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
63-70Subventions
Organisme : NCI NIH HHS
ID : P30 CA008748
Pays : United States
Informations de copyright
© 2022. The Author(s), under exclusive licence to Springer Nature America, Inc.
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