Engineering adeno-associated virus vectors for gene therapy

Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic effects have been achieved for other rare diseases, including haemophilia and Duchenne muscular dystrophy. However, current...

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Bibliographic Details
Published in:	Nature reviews. Genetics Vol. 21; no. 4; pp. 255 - 272
Main Authors:	Li, Chengwen, Samulski, R Jude
Format:	Journal Article
Language:	English
Published:	England Nature Publishing Group 01.04.2020
Subjects:	Adaptive Immunity Blindness Dependovirus - genetics Duchenne's muscular dystrophy Expression vectors Gene therapy Gene transfer Genetic Engineering Genetic Therapy Genetic Vectors - immunology Hemophilia Immune response Immunity, Innate Muscular dystrophy Neuromuscular diseases Rare diseases Spinal muscular atrophy Tropism Vectors (Biology)
ISSN:	1471-0056, 1471-0064, 1471-0064
Online Access:	Get full text
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