Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.
BCL11A
HbS
RNA interference
Sickle cell disease
fetal hemoglobin
gene therapy
hemoglobin switch
hemoglobinopathies
lentiviral vector
shRNAmiR
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:
08
02
2020
accepted:
12
03
2020
entrez:
18
4
2020
pubmed:
18
4
2020
medline:
18
4
2020
Statut:
epublish
Résumé
In this work we provide preclinical data to support initiation of a first-in-human trial for sickle cell disease (SCD) using an approach that relies on reversal of the developmental fetal-to-adult hemoglobin switch. Erythroid-specific knockdown of BCL11A via a lentiviral-encoded microRNA-adapted short hairpin RNA (shRNA
Identifiants
pubmed: 32300607
doi: 10.1016/j.omtm.2020.03.015
pii: S2329-0501(20)30045-0
pmc: PMC7150438
doi:
Types de publication
Journal Article
Langues
eng
Pagination
589-600Informations de copyright
© 2020 The Authors.
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