University of Massachusetts Medical School Faculty Publications
Title
Gene transfer in the liver using recombinant adeno-associated virus
UMMS Affiliation
Gene Therapy Center; Department of Microbiology and Physiological Systems; Department of Pediatrics
Publication Date
2013-05-01
Document Type
Article
Subjects
Dependovirus; Genetic Vectors; Genetic Therapy; Gene Transfer Techniques; Liver
Disciplines
Biomedical Engineering and Bioengineering | Digestive System | Genetic Phenomena | Genetics and Genomics | Hepatology | Microbiology | Molecular Genetics | Therapeutics | Viruses
Abstract
Liver-directed gene transfer and gene therapy are rapidly gaining attention primarily because the liver is centrally involved in a variety of metabolic functions that are affected in various inherited disorders. Recombinant adeno-associated virus (rAAV) is a popular gene delivery vehicle for gene therapy, and intravenous delivery of some rAAV serotypes results in very efficient transduction in the liver. rAAV-mediated gene transfer to the liver can be used to create somatic transgenic animals or disease models for studying the function of various genes and miRNAs. The liver is the target tissue for gene therapy of many inborn metabolic diseases and may also be exploited as a "biofactory" for production of coagulation factors, insulin, growth hormones, and other non-hepatic proteins. Hence, efficient delivery of transgenes and small RNAs to the liver by rAAV vectors has been of long-standing interest to research scientists and clinicians alike. This unit describes methods for delivery of rAAV vectors by several injection routes, followed by a range of analytical methods for assessing the expression, activity, and effects of the transgene and its product. Curr. Protoc. Microbiol. 29:14D.6.1-14D.6.32. (c) 2013 by John Wiley and Sons, Inc.
Keywords
UMCCTS funding, Adeno-associated Virus, Liver, Gene Transfer, Gene therapy
DOI of Published Version
10.1002/9780471729259.mc14d06s29
Source
Curr Protoc Microbiol. 2013 May;Chapter 14:Unit14D.6. doi: 10.1002/9780471729259.mc14d06s29. Link to article on publisher's site
Related Resources
Journal/Book/Conference Title
Current protocols in microbiology
PubMed ID
23686826
Repository Citation
Ahmed SS, Li J, Godwin JG, Gao G, Zhong L. (2013). Gene transfer in the liver using recombinant adeno-associated virus. University of Massachusetts Medical School Faculty Publications. https://doi.org/10.1002/9780471729259.mc14d06s29. Retrieved from https://escholarship.umassmed.edu/faculty_pubs/45
Comments
First author Seemin Seher Ahmed is a doctoral student in the Interdisciplinary Graduate Program in the Graduate School of Biomedical Sciences (GSBS) at UMass Medical School.