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

5-1-2013

Document Type

Article

Subjects

Dependovirus; Genetic Vectors; Genetic Therapy; Gene Transfer Techniques; Liver

Disciplines

Biomedical Engineering and Bioengineering | Genetics and Genomics | Hepatology | Microbiology | Molecular Genetics

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.

Rights and Permissions

Citation: Curr Protoc Microbiol. 2013 May;Chapter 14:Unit14D.6. doi: 10.1002/9780471729259.mc14d06s29. Link to article on publisher's site

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.

Related Resources

Link to Article in PubMed

Keywords

UMCCTS funding

Journal/Book/Conference Title

Current protocols in microbiology

PubMed ID

23686826