.gif)
Investigators publish new data in the report 'Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6.' "Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to administer a therapeutic transgene from the innervated musculature (see also Gene Therapy). With the aim of scaling up this approach to non-human primates, we have injected recombinant adeno-associated vectors (rAAV) serotype 6 expressing enhanced green fluorescent protein (eGFP) into the gastrocnemius muscle of African green monkeys to determine whether this results in efficient transgene delivery to lumbar motor neurons," researchers in Lausanne, Switzerland report.
"Cells expressing eGFP were observed across more than 1 cm of the spinal cord 4 weeks after intramuscular injection, reaching more than half of motor neurons in some cross-sections. Furthermore, quantitative PCR on the spinal cord tissue confirmed that eGFP expression within motor neurons was due to bona fide retrograde transport of the vector genome from the muscle. Although infiltrations of macrophages and lymphocytes were observed in the rAAV2/6-injected muscle, there was no detectable immune response within the transduced region of the spinal cord," wrote C. Towne and colleagues, Ecole polytechnique federale de Lausanne.
The researchers concluded: "These findings imply that retrograde delivery of rAAV serotype 6 in a primate species constitutes a non-invasive and robust approach to transduce motor neurons, a crucial target cell population in neurodegenerative disorders, such as amyotrophic lateral sclerosis and spinal muscular atrophy."
Towne and colleagues published their study in Gene Therapy (Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6. Gene Therapy, 2010;17(1):141-6).
For additional information, contact C. Towne, Brain Mind Institute, Ecole Polytechnique Federale de Lausanne, EPFL, Lausanne, Switzerland.
Publisher contact information for the journal Gene Therapy is: Nature Publishing Group, 345 Park Avenue South, New York, NY 10010-1707, USA.
(c) 2010 Gene Therapy Weekly via NewsRx.com
FIND MORE NEWS-
