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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Summary: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 research indicates that the genetic modification of AAV vectors may further facilitate the success of AAV gene therapy. Vector engineering can increase AAV transduction efficiency (by optimizing the transgene cassette), vector tropism (using capsid engineering) and the ability of the capsid and transgene to avoid the host immune response (by genetically modifying these components), as well as optimize the large-scale production of AAV.
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ISSN:1471-0056
1471-0064
1471-0064
DOI:10.1038/s41576-019-0205-4