Inclusion of a short hairpin RNA targeting <i>BCL11A</i> into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin
Sílvia Pires Lourenço(Children's Hospital of Philadelphia), Stefano Rivella(Children's Hospital of Philadelphia), Virginia Guzikowski(Children's Hospital of Philadelphia), Amaliris Guerra(Children's Hospital of Philadelphia), Valentina Ghiaccio(Children's Hospital of Philadelphia), Yukio Nakamura(Kyorin University), Osheiza Abdulmalik(Children's Hospital of Philadelphia), Laura Breda(Children's Hospital of Philadelphia), Tobias Raabe(University of Pennsylvania), Janet L. Kwiatkowski(Children's Hospital of Philadelphia), Kim Smith‐Whitley(Children's Hospital of Philadelphia), Alexandra U. Zezulin(University of Pennsylvania), Ping La(Children's Hospital of Philadelphia), Danuta Jarocha(Jagiellonian University)
Cited by 10
Related Papers
An atlas of human long non-coding RNAs with accurate 5′ ends
|Nature|2017|1.1k
Establishment of Immortalized Human Erythroid Progenitor Cell Lines Able to Produce Enucleated Red Blood Cells
|PLoS ONE|2013|404
In vivo hematopoietic stem cell modification by mRNA delivery
|Science|2023|321
Dual base editor catalyzes both cytosine and adenine base conversions in human cells
|Nature Biotechnology|2020|294