Paul D. Wellstone Muscular Dystrophy Cooperative Research Center
Projects :: Nationwide Children's Hospital  Wellstone Center
Projects  Project 1: “The implications of Dystrophin-specific T
Cells for DMD Gene Correction”  Proof-of-principle studies in
mouse and dog models of Duchenne muscular dystrophy (DMD) have
established that gene replacement therapy is a promising
treatment strategy.  Attempts to apply the tenets learned from
pre-clinical to clinical protocols did not predict dystrophin-
specific T cells targeting novel epitopes on muscle fibers
downstream of the mutation.  In one case these were expressed on
revertant fibers, a finding contrary to the axiom that forecasts
a tolerizing role for these fibers. Another treatment paradigm,
gentamicin-induced mutation suppression, proved equally
confounding because dystrophin-specific T cells were isolated
from the blood and muscle following treatment.  These
observations require further study to achieve success in gene
correction strategies for DMD.   We can