Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells

To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or at the GATAA motif in the intronic erythroid-specific enhancer, using zinc finger nucleases in human bone marrow (BM)...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Κύριοι συγγραφείς: Kai-Hsin Chang, Sarah E. Smith, Timothy Sullivan, Kai Chen, Qianhe Zhou, Jason A. West, Mei Liu, Yingchun Liu, Benjamin F. Vieira, Chao Sun, Vu P. Hong, Mingxuan Zhang, Xiao Yang, Andreas Reik, Fyodor D. Urnov, Edward J. Rebar, Michael C. Holmes, Olivier Danos, Haiyan Jiang, Siyuan Tan
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: Elsevier 2017-03-01
Σειρά:Molecular Therapy: Methods & Clinical Development
Θέματα:
genome editing
gene therapy
BCL11A
hemoglobinopathies
fetal hemoglobin
fetal globin reactivation
zinc finger nucleases
Διαθέσιμο Online:http://www.sciencedirect.com/science/article/pii/S2329050117300037
Ετικέτες: Προσθήκη ετικέτας
Δεν υπάρχουν, Καταχωρήστε ετικέτα πρώτοι!
Φορτώνει......

Παρόμοια τεκμήρια