Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine
作者机构：Department of Reproductive BiologyAcademic Center for EducationCultureand Research(ACECR)Qom BranchQom ***Iran National Institute of Genetic Engineering and Biotechnology(NIGEB)Tehran 14155-6463Iran Faculty of Interdisciplinary Science and TechnologyTarbiat Modares UniversityTehran 14117-13116Iran Pediatric Cell and Gene Therapy Research CenterGeneCell&Tissue Research InstituteTehran UniversityofMedical SciencesTehran14155-6559Iran Department of NanobiotechnologyFaculty of Biological SciencesTarbiat Modares UniversityTehran 14117-13116Iran Department of BiochemistryFaculty of Biological SciencesTarbiat Modares UniversityTehran 14117-13116Iran
出 版 物：《Genes & Diseases》 (基因与疾病（英文）)
年 卷 期：2024年第11卷第1期
页 面：268-282页
核心收录：
学科分类：0710[理学-生物学] 1006[医学-中西医结合] 1002[医学-临床医学] 07[理学] 08[工学] 09[农学] 071007[理学-遗传学] 0901[农学-作物学] 0836[工学-生物工程] 090102[农学-作物遗传育种] 100602[医学-中西医结合临床] 10[医学]
基 金：Financial support of this work was provided by Chongqing Medical University (China)
主 题：Clinical trials CRISPR/Cas9 Gene therapy Non-viral vectors Viral vectors
摘 要：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.
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