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

• Hoofdauteurs: Mir A Hossain (Auteur), Yong Shen (Auteur), Isaac Knudson (Auteur), Shaleen Thakur (Auteur), Jared R Stees (Auteur), Yi Qiu (Auteur), Betty S Pace (Auteur), Kenneth R Peterson (Auteur), Jörg Bungert (Auteur)
• Formaat: Boek
• Gepubliceerd in: Elsevier, 2016-01-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: 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...