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Robust Long-term Transduction of Common Marmoset Neuromuscular Tissue With rAAV1 and rAAV9
- Source :
- Molecular Therapy. Nucleic Acids, Molecular Therapy: Nucleic Acids, Vol 2, Iss C (2013)
- Publication Year :
- 2013
-
Abstract
- Profiles of recombinant adeno-associated virus (rAAV)-mediated transduction show interspecies differences for each AAV serotype. Robust long-term transgene expression is generally observed in rodents, whereas insufficient transduction is seen in animals with more advanced immune systems. Non-human primates, including the common marmoset, could provide appropriate models for neuromuscular diseases because of their higher brain functions and physiological resemblance to humans. Strategies to induce pathologies in the neuromuscular tissues of non-human primates by rAAV-mediated transduction are promising; however, transgene expression patterns with rAAV transduction have not been elucidated in marmosets. In this study, transduction of adult marmoset skeletal muscle with rAAV9 led to robust and persistent enhanced green fluorescent protein (EGFP) expression that was independent of the muscle fiber type, although lymphocyte infiltration was recognized. Systemic rAAV injection into pregnant marmosets led to transplacental fetal transduction. Surprisingly, the intraperitoneal injection of rAAV1 and rAAV9 into the neonatal marmoset resulted in systemic transduction and persistent transgene expression without lymphocyte infiltration. Skeletal and cardiac muscle were effectively transduced with rAAV1 and rAAV9, respectively. Interestingly, rAAV9 transduction led to intense EGFP signaling in the axons of the corpus callosum. These transduction protocols with rAAV will be useful for investigating gene functions in the neuromuscular tissues and developing gene therapy strategies.Molecular Therapy-Nucleic Acids (2013) 2, e95; doi:10.1038/mtna.2013.21; published online 28 May 2013.
- Subjects :
- Genetic enhancement
Transgene
viruses
lcsh:RM1-950
Cardiac muscle
Skeletal muscle
Marmoset
Methods - Original Article
Biology
Green fluorescent protein
Cell biology
common marmoset
Transduction (genetics)
AAV vector
medicine.anatomical_structure
Immune system
lcsh:Therapeutics. Pharmacology
biology.animal
Drug Discovery
Immunology
medicine
Molecular Medicine
neuromuscular
Subjects
Details
- ISSN :
- 21622531
- Volume :
- 2
- Database :
- OpenAIRE
- Journal :
- Molecular therapy. Nucleic acids
- Accession number :
- edsair.doi.dedup.....66d800818d968d0ff22df1726dd2a0bb