کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
3840230 1247899 2013 14 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Gene therapy for retinal disease
موضوعات مرتبط
علوم پزشکی و سلامت پزشکی و دندانپزشکی پزشکی و دندانپزشکی (عمومی)
پیش نمایش صفحه اول مقاله
Gene therapy for retinal disease
چکیده انگلیسی

Gene therapy strategies for the treatment of inherited retinal diseases have made major advances in recent years. This review focuses on adeno-associated viral (AAV) vector approaches to treat retinal degeneration and, thus, prevent or delay the onset of blindness. Data from human clinical trials of gene therapy for retinal disease show encouraging signs of safety and efficacy from AAV vectors. Recent progress in enhancing cell-specific targeting and transduction efficiency of the various retinal layers plus the use of AAV-delivered growth factors to augment the therapeutic effect and limit cell death suggest even greater success in future human trials is possible.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Translational Research - Volume 161, Issue 4, April 2013, Pages 241–254
نویسندگان
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