UMass Chan Medical School Faculty Publications
Title
Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus
UMMS Affiliation
Gene Therapy Center; Department of Microbiology and Physiological Systems; Department of Pediatrics
Publication Date
2013-02-01
Document Type
Book Chapter
Subjects
Dependovirus; Genetic Vectors; Genetic Therapy; Gene Transfer Techniques; Muscle, Skeletal; Myocardium
Disciplines
Genetics and Genomics | Microbiology | Molecular Genetics
Abstract
Adeno-associated virus (AAV) is a DNA virus with a small ( approximately 4.7 kb) single-stranded genome. It is a naturally replication-defective parvovirus of the dependovirus group. Recombinant AAV (rAAV), for use as a gene transfer vector, is created by replacing the viral rep and cap genes with the transgene of interest along with promoter and polyadenylation sequences. Only the viral inverted terminal repeats (ITRs) are required in cis for replication and packaging during production. The ITRs are also necessary and sufficient for vector genome processing and persistence during transduction. The tissue tropism of the rAAV vector is determined by the AAV capsid. In this unit we will discuss several methods to deliver rAAV in order to transduce cardiac and/or skeletal muscle, including intravenous delivery, intramuscular delivery, isolated limb infusion, intrapericardial injection in neonatal mice, and left ventricular wall injection in adult rats.
Keywords
UMCCTS funding
DOI of Published Version
10.1002/9780471729259.mc14d03s28
Source
Curr Protoc Microbiol. 2013 Feb;Chapter 14:Unit 14D.3. doi: 10.1002/9780471729259.mc14d03s28. Link to article on publisher's site
Related Resources
Journal/Book/Conference Title
Current protocols in microbiology
PubMed ID
23408131
Repository Citation
Gruntman AM, Bish LT, Mueller C, Sweeney H, Flotte TR, Gao G. (2013). Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus. UMass Chan Medical School Faculty Publications. https://doi.org/10.1002/9780471729259.mc14d03s28. Retrieved from https://escholarship.umassmed.edu/faculty_pubs/191