Beta Thalassemia Therapy by CRISPR/Cas۹

سال انتشار: 1400
نوع سند: مقاله کنفرانسی
زبان: انگلیسی
مشاهده: 226

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شناسه ملی سند علمی:

CHGGE01_035

تاریخ نمایه سازی: 22 شهریور 1401

چکیده مقاله:

Beta thalassemia is an inherited blood disorder caused by a deficiency in theβ-globin gene which is considered one of the most important diseases due toits high prevalence. Therefore, treating thalassemia is one of the mostimportant challenges. In this study, we evaluate the genome editing ofthalassemia patients using CRISPR/Cas۹, advantages, and challenges of thistreatment. Articles from the last five years were extracted from GoogleScholar. Databases such as PubMed and Nature were also reviewed.CRISPR (clustered regularly interspaced short palindromic Repeats)/Cas۹ isa modern genetic editing technology. This technology is a compound of smallguide RNAs for identifying the target DNA sequence and Cas۹ protein asnuclease for cutting of DNA.We can reduce the expression of genes related to the production of suppressorprotein for Fetal hemoglobin (BCL۱۱A) and compensate for the deficiency ofHBA with higher fetal hemoglobin by CRISPR/Cas۹. After the sequence ofthe target is cut in the genome by Cas۹, it's completely edited by thehomologous recombination (HR) or repair of non-homologous end joining(NHEJ). Repair of NHEJ following either insertion or deletion will causerestoration while Restoration of HR requires a template DNA molecule tosynthesize another strand using complementary base binding. By usingCRISPR/Cas۹, we can treat thalassemia by regulating the expression of thefetal hemoglobin protein gene along with the increase in HBF production.However, more research is needed because of the possibility of off-targetactivity in CRISPR/Cas۹.

نویسندگان

Nastaran Ghorbanian

Department of Medical Laboratory Sciences, Varastegan Institute for Medical Sciences,Mashhad, Iran.