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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Veröffentlicht in:	Nature reviews. Genetics Jg. 21; H. 4; S. 255 - 272
Hauptverfasser:	Li, Chengwen, Samulski, R Jude
Format:	Journal Article
Sprache:	Englisch
Veröffentlicht:	England Nature Publishing Group 01.04.2020
Schlagworte:	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-Zugang:	Volltext
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