A. Fassati et al., TRANSPLANTATION OF RETROVIRAL PRODUCER CELLS FOR IN-VIVO GENE-TRANSFER INTO MOUSE SKELETAL-MUSCLE, Human gene therapy, 7(5), 1996, pp. 595-602
We describe a new strategy for efficient in vivo gene transfer into sk
eletal muscle using retroviral vectors. Recombinant retroviral produce
r cells, previously treated with the cytostatic drug mitomycin C, were
injected into regenerating muscle of adult nude, nude/mdx, and C57BL/
10 mice. Using LacZ reporter gene activity, we detected efficient tran
sduction in all mouse strains (Nude, mean 11%, range 4.2-21%; C57BL/10
, mean 12 %, range 3,4-20 %; Nude mdx, mean 4.3 %, range 2.1-7 % at 4
weeks post-injection and 6.6%, range 1.3-12 % at 12 weeks post-injecti
on). Foreign gene expression was sustained at high levels for at least
3 months. This strategy allows muscle satellite cells to be transfect
ed in vivo, forming a reservoir of the transgene for incorporation int
o new myofibers in subsequent rounds of degeneration and regeneration.
Because of its efficiency and potentially broad application, this pro
cedure represents a new strategy for in vivo genetic transfer in skele
tal muscle and potentially in other tissues.