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
Descripción
Sumario: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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