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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Zusammenfassung: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