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

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Genes & Diseases 2024年第1期11卷 268-282页

作者： Mohadeseh Khoshandam Hossein Soltaninejad marziyeh mousazadeh Amir Ali Hamidieh Saman Hosseinkhani Department of Reproductive Biology Academic Center for EducationCultureand Research(ACECR)Qom BranchQom ***Iran National Institute of Genetic Engineering and Biotechnology(NIGEB) Tehran 14155-6463Iran Faculty of Interdisciplinary Science and Technology Tarbiat Modares UniversityTehran 14117-13116Iran Pediatric Cell and Gene Therapy Research Center GeneCell&Tissue Research InstituteTehran UniversityofMedical SciencesTehran14155-6559Iran Department of Nanobiotechnology Faculty of Biological SciencesTarbiat Modares UniversityTehran 14117-13116Iran Department of Biochemistry Faculty of Biological SciencesTarbiat Modares UniversityTehran 14117-13116Iran

CRISPR/Cas9 is an effective gene editing tool with broad applications for the pre-vention or treatment of numerous *** depends on CRiSPR(clustered regularly inter-spaced short palindromic repeats)as a bacterial immune system and plays as a gene editing *** 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 ***/Cas9 system has been used to treat cancers through a variety of approaches,with sta-ble gene editing ***,the applications and clinical trials of CRisPR/Cas9 in various illnesses are *** to its high precision and efficiency,CRISPR/Cas9 strategies may treat gene-related illnesses by deleting,inserting,modifying,or blocking the expression of specific *** most challenging barrier to the in vivo use of CRISPR/Cas9 like off-target effects will be *** 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： Clinical trials CRISPR/Cas9 Gene therapy Non-viral vectors Viral vectors

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