UMass Chan Medical School Faculty Publications


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


Document Type

Book Chapter


Dependovirus; Genetic Vectors; Genetic Therapy; Gene Transfer Techniques; Muscle, Skeletal; Myocardium


Genetics and Genomics | Microbiology | Molecular Genetics


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.


UMCCTS funding

DOI of Published Version



Curr Protoc Microbiol. 2013 Feb;Chapter 14:Unit 14D.3. doi: 10.1002/9780471729259.mc14d03s28. Link to article on publisher's site

Related Resources

Link to Article in PubMed

Journal/Book/Conference Title

Current protocols in microbiology

PubMed ID