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Review
. 2023 Mar 25;11(1):268-282.
doi: 10.1016/j.gendis.2023.02.027. eCollection 2024 Jan.

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

Mohadeseh Khoshandam  1   2 , Hossein Soltaninejad  3   4 , Marziyeh Mousazadeh  5 , Amir Ali Hamidieh  4 , Saman Hosseinkhani  6
Affiliations
Review

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

Mohadeseh Khoshandam et al. Genes Dis. .

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.

Keywords: CRISPR/Cas9; Clinical trials; Gene therapy; Non-viral vectors; Viral vectors.

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Figures

Fig. 1
Figure 1
ZFNs, TALENs, and CRISPRs lead to DSBs in the genome and are repaired mainly by two pathways: (i) non-homologous end joining (NHEJ): error-prone; (ii) homology-based repair (HDR).
Fig. 2
Figure 2
CRISPR/Cas9 delivery platforms showing viral and non-viral vectors, plasmid, and RNP.
See this image and copyright information in PMC

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