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

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

doi:10.1016/j.gendis.2023.02.027

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

Review article | 更新时间：2024-02-04

- Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine
- Genes & Diseases Vol. 1, Issue 1, Pages: 268-282(2024)
- 作者机构：
  
  1. Department of Reproductive Biology, Academic Center for Education, Culture, and Research (ACECR), Qom Branch, Qom 371698,Iran,6466
  2. National Institute of Genetic Engineering and Biotechnology (NIGEB), Tehran,Iran,14155-6463
  3. Faculty of Interdisciplinary Science and Technology, Tarbiat Modares University, Tehran 6,Iran,14117-1311
  4. Pediatric Cell and Gene Therapy Research Center, Gene, Cell & Tissue Research Institute, Tehran University of Medical Sciences, Tehran,Iran,14155-6559
  5. Department of Nanobiotechnology, Faculty of Biological Sciences, Tarbiat Modares University, Tehran 6,Iran,14117-1311
  6. Department of Biochemistry, Faculty of Biological Sciences, Tarbiat Modares University, Tehran 6,Iran,14117-1311
- 作者简介：
- 基金信息：
- DOI：10.1016/j.gendis.2023.02.027
  CLC：
- Published：2024，
  
  Published Online：1 January 2024，
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Mohadeseh Khoshandam, Hossein Soltaninejad, Marziyeh Mousazadeh, et al. Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine. [J]. Genes & Diseases 1(1):268-282(2024)
DOI：

Mohadeseh Khoshandam, Hossein Soltaninejad, Marziyeh Mousazadeh, et al. Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine. [J]. Genes & Diseases 1(1):268-282(2024) DOI： 10.1016/j.gendis.2023.02.027.

摘要

Abstract

CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced 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 approaches

it has been broadly investigated to treat numerous hereditary and acquired illnesses

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 stable 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 vectors (such as an Adeno-associated virus (

AAV

))

and the development of non-viral vectors is also considered.

关键词

Keywords

Clinical trialsCRISPR/Cas9Gene therapyNon-viral vectorsViral vectors

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