Khoshandam Mohadeseh, Soltaninejad Hossein, Mousazadeh Marziyeh, Hamidieh Amir Ali, Hosseinkhani Saman
Department of Reproductive Biology, Academic Center for Education, Culture, and Research (ACECR), Qom Branch, Qom 3716986466, Iran.
National Institute of Genetic Engineering and Biotechnology (NIGEB), Tehran 14155-6463, Iran.
Genes Dis. 2023 Mar 25;11(1):268-282. doi: 10.1016/j.gendis.2023.02.027. eCollection 2024 Jan.
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 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 ()), and the development of non-viral vectors is also considered.
CRISPR/Cas9是一种有效的基因编辑工具，在预防或治疗多种疾病方面具有广泛应用。它依赖于作为细菌免疫系统的CRISPR（成簇规律间隔短回文重复序列）并作为一种基因编辑工具发挥作用。由于与其他编辑方法相比，CRISPR/Cas9具有更高的特异性和效率，因此已被广泛研究用于治疗多种遗传性和后天性疾病，包括癌症、溶血性疾病、免疫缺陷疾病、心血管疾病、视觉疾病、神经退行性疾病以及一些X连锁疾病。CRISPR/Cas9系统已通过多种方法和稳定的基因编辑技术用于治疗癌症。在此，将描述CRISPR/Cas9在各种疾病中的应用和临床试验。由于其高精度和高效率，CRISPR/Cas9策略可通过删除、插入、修改或阻断特定基因的表达来治疗基因相关疾病。将讨论使用CRISPR/Cas9时最具挑战性的障碍，如脱靶效应。还将考虑用于CRISPR/Cas9的转染载体的使用，包括病毒载体（如腺相关病毒）以及非病毒载体的开发。
