Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer

L Morandi, P Bernasconi, M Gebbia, M Mora… - Neuromuscular …, 1995 - Elsevier
We report our experience on myoblast transplantation in three Duchenne muscular
dystrophy patients. Pure myoblasts (55× 106 per patient) from HLA-matched donors, were
injected into a tibialis anterior and the controlateral muscle was sham injected. Three
months after transplantation, biopsies from the injected muscles were negative for
dystrophin expression by immunocytochemistry. Reverse transcriptase-PCR (RT-PCR)
failed to amplify any fragments of the deleted regions. This result confirms that myoblast …