Gene Therapy Advances of CRISPR/Cas9 in β-thalassaemia
LI Huan-rong
Abstract：β-thalassaemia is a genetic disease of human β-globin (HBB) gene mutation,there is no cure possible,leading to the disorder of globin expression.CRISPR/Cas system,as the acquired immune system of bacteria and archaea,can transform the genome of many organisms at present.CRISPR/Cas9 systems is widely used for the advantages of easy programming,inexpensive construction,high specificity and multiplexed genome editing.The latest study using CRISPR/Cas9 systems combined with induced pluripotent stem cells to correct human HBB has become possible,meanwhile produces normal hemoglobin to reduce the symptom of patients with β-thalassaemia.The outcome will be a tremendous disasters,if off-target effect occured in human,that's scientist worried about.To increase the safety of CRISPR/Cas9 gene editing,the latest research avoid the off-targets effect by increasing the specificity of CRISPR resection and controlling the Cas9 effectively.We reviewed the latest researches on the CRISPR/Cas9 systems and deal with off target effects.
Keywords：beta-thalassemiaGenesTherapyCRISPR/Cas
Publication Date：2017-01-01
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：4( 185-188 )