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

• Үндсэн зохиолчид: Mir A Hossain (Зохиогч), Yong Shen (Зохиогч), Isaac Knudson (Зохиогч), Shaleen Thakur (Зохиогч), Jared R Stees (Зохиогч), Yi Qiu (Зохиогч), Betty S Pace (Зохиогч), Kenneth R Peterson (Зохиогч), Jörg Bungert (Зохиогч)
• Формат: Ном
• Хэвлэсэн: Elsevier, 2016-01-01T00:00:00Z.
• Нөхцлүүд: article
• Онлайн хандалт: 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...