Paper
0
Highly efficient therapeutic gene editing of human hematopoietic stem cells
Authors
Yuxuan Wu,Jing Zeng
Benjamin Roscoe,Pengpeng Liu,Qiuming Yao,Cícera Lazzarotto,Kendell Clement,Mitchel Cole,Kevin Luk,Cristina Baricordi,Anne Shen,Chunyan Ren,Erica Esrick,John Manis,David Dorfman,David Williams,Alessandra Biffi,Carlo Brugnara,Luca Biasco,Christian Brendel,Luca Pinello,Shengdar Tsai,Scot Wolfe
+21 authors
,Daniel BauerJournal
Published
Mar 25, 2019
Show more
Save
Tip0
TipSave
Abstract
Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by induction of fetal hemoglobin (HbF, α
Paper PDF
This PDF hasn't been uploaded yet.
Do not upload any copyrighted content to the site, only open-access content.
or