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Clinical Applications of the Crispr/Cas9 Genome-Editing System: Delivery Options and Challenges in Precision Medicine Publisher



Khoshandam M1, 2 ; Soltaninejad H3, 4 ; Mousazadeh M5 ; Hamidieh AA4 ; Hosseinkhani S6
Authors
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Authors Affiliations
  1. 1. Department of Reproductive Biology, Academic Center for Education, Culture, and Research (ACECR), Qom Branch, Qom, 3716986466, Iran
  2. 2. National Institute of Genetic Engineering and Biotechnology (NIGEB), Tehran, 14155-6463, Iran
  3. 3. Faculty of Interdisciplinary Science and Technology, Tarbiat Modares University, Tehran, 14117-13116, Iran
  4. 4. Pediatric Cell and Gene Therapy Research Center, Gene, Cell & Tissue Research Institute, Tehran University of Medical Sciences, Tehran, 14155-6559, Iran
  5. 5. Department of Nanobiotechnology, Faculty of Biological Sciences, Tarbiat Modares University, Tehran, 14117-13116, Iran
  6. 6. Department of Biochemistry, Faculty of Biological Sciences, Tarbiat Modares University, Tehran, 14117-13116, Iran

Source: Genes and Diseases Published:2024


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. © 2023 Chongqing Medical University
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