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Highly Efficient Therapeutic Gene Editing of BCL11A Enhancer in Human Hematopoietic Stem Cells from SS-Hemoglobinopathy Patients for Fetal Hemoglobin Induction

Jing Zeng,Daniel E. Bauer,Yuxuan Wu,Benjamin P. Roscoe,Pengpeng Liu,Qiuming Yao,Cicera Lazzarrotto,M. Kendell Clement,Mitchel A. Cole,Kevin Luk,Cristina Baricordi,Erica B. Esrick,John P. Manis,David Dorfma,David A. Williams,Alessandra Biffi,Carlo Brugnara,Luca Biasco,Christian Brendel,Luca Pinello,Shengdar Tsai,Scot A. Wolfe

Blood（2018）

Cited 2|Views14
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Key words
Gene Editing,siRNA Therapeutics,Multiplex Genome Editing,Gene Therapy
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