首页|Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies

Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies

扫码查看
原文链接
  • 国家科技期刊平台
  • NETL
  • NSTL
  • 万方数据
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.

sickle cell diseasesickle cell anemiafetal hemoglobinhemoglobinopathyCRISPRgene editinggenome engineering

James B.Papizan、Shaina N.Porter、Akshay Sharma、Shondra M.Pruett-Miller

展开 >

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, USA

Department of Bone Marrow Transplantation and Cellular Therapy, St.Jude Children's Research Hospital, Memphis, TN 38105, USA

2021

生物医学研究杂志(英文版)
南京医科大学

生物医学研究杂志(英文版)

CSCD
影响因子：0.794
ISSN：1674-8301
年,卷(期)：2021.35(2)
  • 179