Potential therapeutic application of CRISPR-Cas9 in Huntington s disease

Publish Year: 1396
نوع سند: مقاله کنفرانسی
زبان: English
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شناسه ملی سند علمی:

NGCMED10_148

تاریخ نمایه سازی: 16 تیر 1397

Abstract:

Backgraound: Huntington s disease caused by a extension of a CAG repeat in huntingtin gene and lead to anelongated polyglutamine tract that is toxic for the brain. So far, the drugs available for this disorder aresymptomatic treatment; unfortunately, they are not successful in curing the disease or slow down the progressionof the disease. But CRISPR/Cas9 recently has been used as an constant treatment. So, the purpose of this studywas to evaluation articles in this field.Methods: Searching for related publications was done using keywords crispr-cas9 and Huntington s disease in PubMed, Scopus and Web of Science databases.Results: The rapid growth of the CRISPR-Cas9-mediated genome editing tool has revolutionized the field ofgene therapy. One of the application this procedure is gene therapy in various diseases like neuromusculardisorders and Huntington s disease. Currently, decrease of HTT mRNA levels with this method is a therapeuticchoice for HD. The major therapeutic profit is expected when the mutant allele is entirely silenced whileexpression from the normal allele is left intact. In addition to therapeutic benefits , this method is also used tomake genetically modified animal models like pig for neurodegenerative disease in order to enhancement insightabout diagnosis, remedy, and prevention of neurodegenerative disorders.Conclusion: Our research provide proof of Therapeutic characteristic of crispr-cas9 in Huntington s diseasetherapy Although CRISPR/Cas9 system has many problems to be solved, such as off-target effects, deliverysystem, efficacy, safety concerns, but is being practical in biotechnology and clinical trial.

Authors

Maliehe Alimardani

Department of Medical Genetics, Tabriz University of Medical Sciences, Tabriz, Iran- Department of Medical Genetics, Faculty of Medicine,Mashhad University of Medical Sciences, Mashhad,Iran- Student Research Committee, Faculty of Medicine, Mashhad Univers

Mahsa Farjami

Student Research Committee, Faculty of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran

Sima Mansoori Derakhshan

Department of Medical Genetics, Tabriz University of Medical Sciences, Tabriz, Iran

Majid Mojarrad

Department of Medical Genetics, Faculty of Medicine,Mashhad University of Medical Sciences, Mashhad,Iran