موانع و محدودیت‌های کلینیکی انجام Small interfering RNA delivery (siRNA delivery) بر پایه‌ی وکتورهای غیر ویروسی

نوع مقاله : مقاله مروری

نویسندگان

1 کارشناس ارشد، گروه ژنتیک و بیولوژی مولکولی، دانشکده‌ی پزشکی، دانشگاه علوم پزشکی اصفهان، اصفهان، ایران

2 استادیار، گروه ژنتیک و بیولوژی مولکولی، دانشکده‌ی پزشکی، دانشگاه علوم پزشکی اصفهان، اصفهان، ایران

چکیده

در سال‌های اخیر، ژن درمانی از طریق siRNA (Small interfering RNA) توجهات زیادی را معطوف خود ساخته است. این پدیده بر اساس PTGS (Post transcriptional gene silencing) استوار است. به دلیل نقایص وکتورهای ویروسی، استفاده از وکتورهای Non viral برای انتقال نوکلئیک اسید مورد توجه قرار گرفته است. درست است که وکتورهای Non viral نسبت به انواع ویروسی، میزان ترانسفکشن کمی دارند، اما Safety وکتورهای غیر ویروسی بسیار بیشتر می‌باشد. قابلیت‌های کلیدی siRNA مثل انطباق پذیری بالا، کاربرد در دوزهای کم و همه کاره بودن آن، سبب استفاده از siRNA در روش‌های انتقال ژن شده است. با این حال، دارای نقایصی مثل تحریک سیستم ایمنی و Off target silencing نیز می‌باشد. در این مقاله، بیشتر سعی بر این بود تا مشکلاتی که در سر راه انتقال siRNA به سلول وجود دارد، بررسی شود. با توجه به اطلاعاتی که در دست است، گفته می‌شود که پیشرفت هر چه بیشتر siRNA delivery می‌تواند در آینده به یک روش قابل اتکا جهت درمان بیماری‌هایی که پایه‌ی ژنتیکی دارند، مبدل گردد. 

کلیدواژه‌ها

عنوان مقاله [English]

Limitations of Clinical Application of siRNA Delivery Based on Non-Viral Vectors

نویسندگان [English]

  • Reza Ghavimi 1
  • Meraj Pourhossein 2
1 Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran
2 Assistant Professor, Department of Genetics and Molecular Biology, School of Medicine, Isfahan University of Medical Sciences, Isfahan, Iran
چکیده [English]

In recent years, much more attentions have been focused on siRNA-based gene therapy. This approach is based on PTGS (Post-transcriptional gene silencing). Due to some defects in viral vectors, non-viral vectors have been used to deliver nucleic acids into target cells. Although, transfection efficiency in non-viral vectors is less than viral ones, but safety of non-viral vectors is much more. Characteristic features of siRNA, such as high compatibility, application in low doses and its versatility make it suitable in the gene therapy field. However, some challenges, such as stimulating immune system and Off-target silencing will be remain. In this review article, we express bottlenecks existing in siRNA delivery into target cells. According to the information, with further development of siRNA delivery in the future, it could be a promising approach in treatment for a variety of genetic diseases.

کلیدواژه‌ها [English]

  • siRNA delivery
  • Non viral vector
  • Gene therapy

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مجله دانشکده پزشکی اصفهان
دوره 32، شماره 272 - شماره پیاپی 272
فروردین و اردیبهشت 1393
صفحه 34-49

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سابقه مقاله

  • تاریخ دریافت: 07 مهر 1392
  • تاریخ بازنگری: 11 آبان 1403
  • تاریخ پذیرش: 17 فروردین 1401

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