成簇的正则间隔短联合重复序列--基因组技术的新时代及其在治疗学中的应用

IF 0.2 Q4 MULTIDISCIPLINARY SCIENCES Suranaree Journal of Science and Technology Pub Date : 2023-12-13 DOI:10.55766/sujst-2023-04-e0229
Karthik Rajaram mohan, Saramma Mathew Fenn, Ramachandra Reddy
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引用次数: 0

摘要

簇状有规律间隔短链重复序列(Clustered Regularly Interspaced Short Palindromic Repeats)被称为CRISPR。细菌的基因组序列重复序列之间有脱氧核糖核酸的 "间隔 "序列,与病毒的基因组序列相似,被发现具有重复的 DNA 序列,称为 CRISPR。基因工程中的 CRISPR 技术为医学领域带来了革命性的变化,它可以治疗各种难以治疗的与基因有关的疾病,如先天性无脑畸形(Lebers Congenital amaurosis),其中线粒体 DNA MT-ND4(NADH ubiquinone oxidoreductase chain4)1178G 基因突变导致双等位基因缺失,通过玻璃体内注射 CRISPR-Cas9 基因组编辑技术产生的 AAV2-ND4 可以治疗这种疾病。CRISPR-Cas9 基因组编辑技术用于去除导致视网膜色素变性的受体增强蛋白-6(Reep6 p.Leu135Pro )基因的表达。利用CRISPR-Cas9基因组编辑技术,有效清除了由肌球蛋白(MYOR)基因突变引起的开角型青光眼。通过将 Cas9/sgRNA 注入角膜基质,缓解了导致梅斯曼上皮性角膜营养不良症(MECD)的 KRT-12 基因突变。CRISPR-Cas9基因组测序技术被用于治疗血友病-B,这是一种由因子IX基因Y371D突变引起的遗传性疾病。
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Clustered Regularly Interspaced Short Palindromic Repeats- a new era in Genomic technology and its applications in Theranostics
Clustered Regularly Interspaced Short Palindromic Repeats is termed as CRISPR. Bacteria having "spacer" sequences of deoxyribonucleic acid between the repeats of genomic sequences that resemble genomic sequences in viruses were found to have repetitive DNA sequences known as CRISPR.The CRISPR technology in genetic engineering has revolutionalized the field of medicine in treating various genetically linked diseases that are difficult to treat such as Lebers Congenital amaurosis, in which there is bi-allelic deletion caused by mutation in mitochondrial DNA MT-ND4(NADH ubiquinone oxidoreductase chain4) 1178G are treated by intravitreous injection of AAV2-ND4 produced by CRISPR-Cas9 genomic editing technology.CRISPR-Cas9 genomic editing is used to remove the expression of receptor Enhancer Protein-6 (Reep6 p.Leu135Pro ) gene that causes retinitis pigmentosa. Open angle Glaucoma caused by mutations in myocilin (MYOR) gene was effectively removed out by CRISPR-Cas9 editing technology. Mutation in KRT-12 gene that caused Meesman epithelial corneal dystrophy ( MECD) was alleviated by Cas9/sgRNA injection into the stroma of cornea. CRISPR-Cas9 genomic sequencing used in the treatment of Haemophilia -B , an inherited disease caused by mutation of factor IX gene Y371D was successfully modified by CRISPR-
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Suranaree Journal of Science and Technology
Suranaree Journal of Science and Technology MULTIDISCIPLINARY SCIENCES-
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50.00%
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