Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy

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 sho...

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Publicado en:Mol Ther Methods Clin Dev
Main Authors: Brendel, Christian, Negre, Olivier, Rothe, Michael, Guda, Swaroopa, Parsons, Geoff, Harris, Chad, McGuinness, Meaghan, Abriss, Daniela, Tsytsykova, Alla, Klatt, Denise, Bentler, Martin, Pellin, Danilo, Christiansen, Lauryn, Schambach, Axel, Manis, John, Trebeden-Negre, Helene, Bonner, Melissa, Esrick, Erica, Veres, Gabor, Armant, Myriam, Williams, David A.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7150438/
https://ncbi.nlm.nih.gov/pubmed/32300607
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.03.015
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