Therapeutic gene editing strategies using CRISPR-Cas9 for theβ-hemoglobinopathies

作     者：James B.Papizan Shaina N.Porter Akshay Sharma Shondra M.Pruett-Miller James B.Papizan;Shaina N.Porter;Akshay Sharma;Shondra M.Pruett-Miller

作者机构：Department of Cellular and Molecular BiologySt.Jude Children's Research HospitalMemphisTN 38105USA Center for Advanced Genome EngineeringSt.Jude Children's Research HospitalMemphisTN 38105USA Department of Bone Marrow Transplantation and Cellular TherapySt.Jude Children's Research HospitalMemphisTN 38105USA 

出 版 物：《The Journal of Biomedical Research》 (生物医学研究杂志（英文版）)

年 卷 期：2021年第35卷第2期

页      面：115-134页

核心收录：

学科分类：0710[理学-生物学] 07[理学] 08[工学] 09[农学] 071007[理学-遗传学] 0901[农学-作物学] 0836[工学-生物工程] 090102[农学-作物遗传育种] 

主　　题：sickle cell disease sickle cell anemia fetal hemoglobin hemoglobinopathy CRISPR gene editing genome engineering 

摘      要：With advancements in gene editing technologies,our ability to make precise and efficient modifications to the genome is increasing at a remarkable rate,paving the way for scientists and clinicians to uniquely treat a multitude of previously irremediable ***-Cas9,short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9,is a gene editing platform with the ability to alter the nucleotide sequence of the genome in living *** technology is increasing the number and pace at which new gene editing treatments for genetic disorders are moving toward the ***β-hemoglobinopathies are a group of monogenic diseases,which despite their high prevalence and chronic debilitating nature,continue to have few therapeutic options *** this review,we will discuss our existing comprehension of the genetics and current state of treatment forβ-hemoglobinopathies,consider potential genome editing therapeutic strategies,and provide an overview of the current state of clinical trials using CRISPR-Cas9 gene editing.