Adeno-associated viral vector targeted evolution for neurofibromatosis gene delivery.

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Tác giả: Nicholas M Boulis, Ana Carneiro, Brett Henshey, Kecheng Lei, David Schaffer

Ngôn ngữ: eng

Ký hiệu phân loại: 336.09 Historical, geographic, persons treatment; associations of sovereign states

Thông tin xuất bản: England : Trends in molecular medicine , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 91040

Neurofibromatosis type 1 (NF1) is an inherited genetic disease resulting from pathogenic mutations in NF1 that drive tumor formation along peripheral nerves, leading to many functional consequences. Tumor removal or treatment often results in regrowth and/or nerve damage. Addressing NF1 pathogenic variations at the cellular level through gene therapy holds great potential for long-term treatment of patients with NF1. Adeno-associated viruses (AAVs) are broadly used gene delivery vehicles for gene therapies because of their low pathogenicity, ability to transduce nondividing cells, and potential for long-term gene expression. This article explores the landscape of AAV-mediated gene delivery strategies for NF1, discusses the challenges of efficient delivery to relevant cell types, and highlights the progress in vector design strategies.
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