Abstract
<jats:title>Abstract</jats:title> <jats:sec> <jats:label/> <jats:p> Beta‐thalassemia is a genetic disorder caused by various pathogenic variants in the <jats:italic>HBB</jats:italic> gene cluster and downregulation of β‐globin production. Conventional treatments for intermediate and severe forms of beta‐thalassemia include regular red blood cell transfusion, splenectomy and allogeneic hematopoietic stem cell transplantation (HSCT). Blood transfusion is safe, but long‐term blood transfusion may cause iron overload, resulting in endocrine complications, and iron chelation therapy is required. Concerning allogeneic HSCT, a human lymphocyte antigen (HLA)‐matched donor is rare, and acute graft versus host disease (GVHD) may ensue. The site‐specific RNA protein interaction makes CRISPR (clustered regularly interspaced short palindromic repeats)/Cas9 (CRISPR‐associated protein 9) a popular gene editing tool because of its simplicity for guide RNA design and high editing efficiency. Therefore, genome editing of autogeneic HSCT by CRISPR/Cas systems sheds light on beta‐thalassemia therapies, which may eventually lead to a cure, besides avoiding GVHD. </jats:p> </jats:sec> <jats:sec> <jats:title>Key Concepts</jats:title> <jats:p> <jats:list list-type="bullet"> <jats:list-item> <jats:p>Overview of β‐thalassemia and its treatment.</jats:p> </jats:list-item> <jats:list-item> <jats:p>CRISPR/Cas systems and related derivative technologies.</jats:p> </jats:list-item> <jats:list-item> <jats:p>CRISPR/Cas9 targeting cell candidates for β‐thalassemia therapy.</jats:p> </jats:list-item> <jats:list-item> <jats:p>Target genes edited by CRISPR/Cas‐based technology for β‐thalassemia therapy.</jats:p> </jats:list-item> <jats:list-item> <jats:p>Clinical trials on β‐thalassemia with CRISPR‐associated technology.</jats:p> </jats:list-item> <jats:list-item> <jats:p>Advantages and limitations of CRISPR/Cas technology.</jats:p> </jats:list-item> </jats:list> </jats:p> </jats:sec>