Activation of Fetal γ-globin Gene Expression via Direct Protein Delivery of Synthetic Zinc-finger DNA-Binding Domains

• Main Authors: Mir A Hossain (Author), Yong Shen (Author), Isaac Knudson (Author), Shaleen Thakur (Author), Jared R Stees (Author), Yi Qiu (Author), Betty S Pace (Author), Kenneth R Peterson (Author), Jörg Bungert (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Reactivation of γ-globin expression has been shown to ameliorate disease phenotypes associated with mutations in the adult β-globin gene, including sickle cell disease. Specific mutations in the promoter of the γ-globin genes are known to prevent repression of the genes in the adult and thus lead to...