کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
5433890 1509008 2016 16 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9
موضوعات مرتبط
مهندسی و علوم پایه مهندسی مواد بیومتریال
پیش نمایش صفحه اول مقاله
Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9
چکیده انگلیسی

Several attempts have been made to discover the ideal vector for gene therapy in central nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle since they exhibit stable transgene expression in post-mitotic cells, neuronal tropism, low risk of insertional mutagenesis and diminished immune responses. Additionally, the discovery that a particular serotype, AAV9, bypasses the blood-brain barrier has raised the possibility of intravascular administration as a non-invasive delivery route to achieve widespread CNS gene expression. AAV9 intravenous delivery has already shown promising results for several diseases in animal models, including lysosomal storage disorders and motor neuron diseases, opening the way to the first clinical trial in the field. This review presents an overview of clinical trials for CNS disorders using AAVs and will focus on preclinical studies based on the systemic gene delivery using AAV9.

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ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Journal of Controlled Release - Volume 241, 10 November 2016, Pages 94-109
نویسندگان
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