Me. Shy et al., AN ADENOVIRAL VECTOR CAN TRANSFER LACZ EXPRESSION INTO SCHWANN-CELLS IN CULTURE AND IN SCIATIC-NERVE, Annals of neurology, 38(3), 1995, pp. 429-436
Although a number of genetic defects in the P0, peripheral myelin prot
ein-22, and connexin-32 genes recently were shown to cause the demyeli
nating forms of Charcot-Marie-Tooth disease, there is yet no effective
treatment for these patients. Recent studies showed that replication
defective adenoviral vectors can efficiently introduce genes into musc
le, brain, lung, and other tissues, suggesting that this vector system
may be useful for the treatment of a number of genetic diseases. In t
his work, we demonstrated that a replication deficient adenovirus expr
essing the Escherichia coli beta-galactosidase gene (AdCMVLacZ) can in
troduce genes into Schwann cells, in culture as well as in sciatic ner
ve. Schwann cells cultured at a multiplicity of infection of 250:1 did
not demonstrate cytopathic effects. Following injection of AdCMVLacZ
into sciatic nerve of rats, lacZ-expressing, myelinating Schwann cells
could be detected for at least 45 days. These data suggest that in th
e future, these vectors may be useful both in perturbing Schwann cell
gene expression and in designing therapies for the treatment of Charco
t-Marie-Tooth disease.