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 |
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| Hauptverfasser: | , |
| Format: | Journal Article |
| Sprache: | Englisch |
| Veröffentlicht: |
England
Nature Publishing Group
01.04.2020
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| ISSN: | 1471-0056, 1471-0064, 1471-0064 |
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| Abstract | 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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| AbstractList | 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.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. 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.Adeno-associated virus (AAV) vector-mediated gene delivery has had long-term therapeutic effects for several diseases, including haemophilia and Duchenne muscular dystrophy. Genetically modifying AAV vectors to increase their transduction efficiency, vector tropism and ability to avoid the host immune response may further increase the success of AAV 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 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. |
| Author | Samulski, R Jude Li, Chengwen |
| Author_xml | – sequence: 1 givenname: Chengwen surname: Li fullname: Li, Chengwen email: chengwen@med.unc.edu, chengwen@med.unc.edu organization: Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. chengwen@med.unc.edu – sequence: 2 givenname: R Jude surname: Samulski fullname: Samulski, R Jude email: rjs@med.unc.edu, rjs@med.unc.edu organization: Department of Pharmacology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. rjs@med.unc.edu |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/32042148$$D View this record in MEDLINE/PubMed |
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| ContentType | Journal Article |
| Copyright | 2020© Springer Nature Limited 2020 Springer Nature Limited 2020. |
| Copyright_xml | – notice: 2020© Springer Nature Limited 2020 – notice: Springer Nature Limited 2020. |
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| Snippet | Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the treatment of inherited blindness and spinal muscular atrophy, and... |
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| SubjectTerms | 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) |
| Title | Engineering adeno-associated virus vectors for gene therapy |
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