Ontology highlight
ABSTRACT:
SUBMITTER: Xiong Z
PROVIDER: S-EPMC6850948 | biostudies-literature | 2019 Dec
REPOSITORIES: biostudies-literature
Xiong Zeyu Z Xie Yingjun Y Yang Yi Y Xue Yanting Y Wang Ding D Lin Shouheng S Chen Diyu D Lu Dian D He Lina L Song Bing B Yang Yinghong Y Sun Xiaofang X
Journal of cellular and molecular medicine 20191021 12
β-thalassaemia is a prevalent hereditary haematological disease caused by mutations in the human haemoglobin β (HBB) gene. Among them, the HBB IVS2-654 (C > T) mutation, which is in the intron, creates an aberrant splicing site. Bone marrow transplantation for curing β-thalassaemia is limited due to the lack of matched donors. The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), as a widely used tool for gene editing, is able to target specif ...[more]