One-Step Biallelic and Scarless Correction of a β-Thalassemia Mutation in Patient-Specific iPSCs without Drug Selection

• Main Authors: Yali Liu (Author), Yi Yang (Author), Xiangjin Kang (Author), Bin Lin (Author), Qian Yu (Author), Bing Song (Author), Ge Gao (Author), Yaoyong Chen (Author), Xiaofang Sun (Author), Xiaoping Li (Author), Lei Bu (Author), Yong Fan (Author)
• Formato: Livro
• Publicado em: Elsevier, 2017-03-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Monogenic disorders (MGDs), which are caused by single gene mutations, have a serious effect on human health. Among these, β-thalassemia (β-thal) represents one of the most common hereditary hematological diseases caused by mutations in the human hemoglobin β (HBB) gene. The technologies of induced...