首页|Clinical applications of the CRISPR/Cas9 genome-editing system:Delivery options and challenges in precision medicine

Clinical applications of the CRISPR/Cas9 genome-editing system:Delivery options and challenges in precision medicine

扫码查看
原文链接
  • NETL
  • NSTL
  • 万方数据
CRISPR/Cas9 is an effective gene editing tool with broad applications for the pre-vention or treatment of numerous diseases.It depends on CRISPR(clustered regularly inter-spaced short palindromic repeats)as a bacterial immune system and plays as a gene editing tool.Due to the higher specificity and efficiency of CRISPR/Cas9 compared to other editing ap-proaches,it has been broadly investigated to treat numerous hereditary and acquired ill-nesses,including cancers,hemolytic diseases,immunodeficiency disorders,cardiovascular diseases,visual maladies,neurodegenerative conditions,and a few X-linked disorders.CRISPR/Cas9 system has been used to treat cancers through a variety of approaches,with sta-ble gene editing techniques.Here,the applications and clinical trials of CRISPR/Cas9 in various illnesses are described.Due to its high precision and efficiency,CRISPR/Cas9 strategies may treat gene-related illnesses by deleting,inserting,modifying,or blocking the expression of specific genes.The most challenging barrier to the in vivo use of CRISPR/Cas9 like off-target effects will be discussed.The use of transfection vehicles for CRISPR/Cas9,including viral vec-tors(such as an Adeno-associated virus(AAV)),and the development of non-viral vectors is also considered.

Clinical trialsCRISPR/Cas9Gene therapyNon-viral vectorsViral vectors

Mohadeseh Khoshandam、Hossein Soltaninejad、Marziyeh Mousazadeh、Amir Ali Hamidieh、Saman Hosseinkhani

展开 >

Department of Reproductive Biology,Academic Center for Education,Culture,and Research(ACECR),Qom Branch,Qom 3716986466,Iran

National Institute of Genetic Engineering and Biotechnology(NIGEB),Tehran 14155-6463,Iran

Faculty of Interdisciplinary Science and Technology,Tarbiat Modares University,Tehran 14117-13116,Iran

Pediatric Cell and Gene Therapy Research Center,Gene,Cell & Tissue Research Institute,Tehran University of Medical Sciences,Tehran 14155-6559,Iran

Department of Nanobiotechnology,Faculty of Biological Sciences,Tarbiat Modares University,Tehran 14117-13116,Iran

Department of Biochemistry,Faculty of Biological Sciences,Tarbiat Modares University,Tehran 14117-13116,Iran

展开 >

Chongqing Medical University(China)

2024

10.1016/j.gendis.2023.02.027

基因与疾病(英文)

基因与疾病(英文)

ISSN：
年,卷(期)：2024.11(1)
  • 154