Adeno-Associated Virus Gene Therapy for Liver Disease

The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made rema...

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Detalles Bibliográficos
Autores principales: Kattenhorn, Lisa M., Tipper, Christopher H., Stoica, Lorelei, Geraghty, Deborah S., Wright, Teresa L., Clark, K. Reed, Wadsworth, Samuel C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2016
Materias:
Review Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5177998/
https://www.ncbi.nlm.nih.gov/pubmed/27897038
http://dx.doi.org/10.1089/hum.2016.160
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author Kattenhorn, Lisa M.
Tipper, Christopher H.
Stoica, Lorelei
Geraghty, Deborah S.
Wright, Teresa L.
Clark, K. Reed
Wadsworth, Samuel C.
author_facet Kattenhorn, Lisa M.
Tipper, Christopher H.
Stoica, Lorelei
Geraghty, Deborah S.
Wright, Teresa L.
Clark, K. Reed
Wadsworth, Samuel C.
author_sort Kattenhorn, Lisa M.
collection PubMed
description The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made remarkable strides, with a number of clinical trials currently planned and ongoing in hemophilia A and B, as well as other liver disorders. This review focuses on liver-directed AAV gene therapy, including historic context, current challenges, and future developments.
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spelling pubmed-51779982017-01-11 Adeno-Associated Virus Gene Therapy for Liver Disease Kattenhorn, Lisa M. Tipper, Christopher H. Stoica, Lorelei Geraghty, Deborah S. Wright, Teresa L. Clark, K. Reed Wadsworth, Samuel C. Hum Gene Ther Review Articles The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made remarkable strides, with a number of clinical trials currently planned and ongoing in hemophilia A and B, as well as other liver disorders. This review focuses on liver-directed AAV gene therapy, including historic context, current challenges, and future developments. Mary Ann Liebert, Inc. 2016-12-01 2016-12-01 /pmc/articles/PMC5177998/ /pubmed/27897038 http://dx.doi.org/10.1089/hum.2016.160 Text en © Lisa M. Kattenhorn, et al., 2016; Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons Attribution Noncommercial License (http://creativecommons.org/licenses/by-nc/4.0/) which permits any noncommercial use, distribution, and reproduction in any medium, provided the original author(s) and the source are credited.
spellingShingle Review Articles
Kattenhorn, Lisa M.
Tipper, Christopher H.
Stoica, Lorelei
Geraghty, Deborah S.
Wright, Teresa L.
Clark, K. Reed
Wadsworth, Samuel C.
Adeno-Associated Virus Gene Therapy for Liver Disease
title Adeno-Associated Virus Gene Therapy for Liver Disease
title_full Adeno-Associated Virus Gene Therapy for Liver Disease
title_fullStr Adeno-Associated Virus Gene Therapy for Liver Disease
title_full_unstemmed Adeno-Associated Virus Gene Therapy for Liver Disease
title_short Adeno-Associated Virus Gene Therapy for Liver Disease
title_sort adeno-associated virus gene therapy for liver disease
topic Review Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5177998/
https://www.ncbi.nlm.nih.gov/pubmed/27897038
http://dx.doi.org/10.1089/hum.2016.160
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