کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
5434064 1398084 2016 15 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Delivery methods for site-specific nucleases: Achieving the full potential of therapeutic gene editing
ترجمه فارسی عنوان
روش های تحویل برای هسته های خاص سایت: دستیابی به پتانسیل کامل از ویرایش ژنتیک درمانی
موضوعات مرتبط
مهندسی و علوم پایه مهندسی مواد بیومتریال
چکیده انگلیسی

The advent of site-specific nucleases, particularly CRISPR/Cas9, provides researchers with the unprecedented ability to manipulate genomic sequences. These nucleases are used to create model cell lines, engineer metabolic pathways, produce transgenic animals and plants, perform genome-wide functional screen and, most importantly, treat human diseases that are difficult to tackle by traditional medications. Considerable efforts have been devoted to improving the efficiency and specificity of nucleases for clinical applications. However, safe and efficient delivery methods remain the major obstacle for therapeutic gene editing. In this review, we summarize the recent progress on nuclease delivery methods, highlight their impact on the outcomes of gene editing and discuss the potential of different delivery approaches for therapeutic gene editing.

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ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Journal of Controlled Release - Volume 244, Part A, 28 December 2016, Pages 83-97
نویسندگان
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