In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

• Main Authors: Sowmya Pattabhi (Author), Samantha N. Lotti (Author), Mason P. Berger (Author), Swati Singh (Author), Christopher T. Lux (Author), Kyle Jacoby (Author), Calvin Lee (Author), Olivier Negre (Author), Andrew M. Scharenberg (Author), David J. Rawlings (Author)
• Format: Book
• Published: Elsevier, 2019-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit precise homology-directed repair (HDR) while concurre...