Efficient and in situ correction of hemoglobin Constant Spring mutation by prime editing in human hematopoietic cells

• Principais autores: Congwen Shao (Autor), Qing Liu (Autor), Jinchao Xu (Autor), Jianxiang Zhang (Autor), Chengpeng Zhang (Autor), Ye Xin (Autor), Yuhua Ye (Autor), Bin Lin (Autor), Xinhua Zhang (Autor), Li Cheng (Autor), Xiangmin Xu (Autor), Peng Xu (Autor)
• Formato: Livro
• Publicado em: Elsevier, 2024-12-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Hemoglobin Constant Spring (Hb CS) is the most common non-deletional and clinically significant α-thalassemic mutation, and it is caused by an anti-termination mutation at the α2-globin gene stop codon. We developed a prime editing strategy for the creation and correction of Hb CS. We showed that pr...