Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies
James B.Papizan1
Shaina N.Porter1
Akshay Sharma2
Shondra M.Pruett-Miller1
1.Department of Cellular and Molecular Biology, St.Jude Children's Research Hospital, Memphis, TN 38105, USA;Center for Advanced Genome Engineering, St.Jude Children's Research Hospital, Memphis, TN 38105, USA2.Department of Bone Marrow Transplantation and Cellular Therapy, St.Jude Children's Research Hospital, Memphis, TN 38105, USA
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 diseases.CRISPR-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 cells.This technology is increasing the number and pace at which new gene editing treatments for genetic disorders are moving toward the clinic.The β-hemoglobinopathies are a group of monogenic diseases,which despite their high prevalence and chronic debilitating nature,continue to have few therapeutic options available.In this review,we will discuss our existing comprehension of the genetics and current state of treatment for β-hemoglobinopathies,consider potential genome editing therapeutic strategies,and provide an overview of the current state of clinical trials using CRISPR-Cas9 gene editing.
机标关键词：
分类号：Q789(基因工程（遗传工程）)
论文发表日期：2021-03-30
在线出版日期：2025-08-15（本平台首次上网日期，不代表文献的发表时间）
页数：20( 115-134 )
英文信息