查看文章

© The American Society of Gene Therapy commentary species, probably as a result of differential expression of receptors across species. For instance, direct administration of rAAV5 into the marmoset brain resulted in consistent detection of transduced astrocytes, albeit a minority compared with neurons, whereas injection of the same viral preparation into the rodent brain infected only neurons. 25, 26 In addition, variability in transduction pattern and efficiency could be due to the method of viral purification, which may lead to impurities in the vector preparation that may act as carrier proteins. However, in this study Foust et al. 5 attempted to control for this issue. The clinical relevance of the recent findings of Foust et al. 5 is considerable. Efficiently targeting the spinal cord with rAAV to treat or study motor neuron diseases such as spinal muscular atrophy and amyotrophic lateral sclerosis is a direct implication of these …