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

L. Morandi, P. Bernasconi, M. Gebbia, M. Mora, F. Crosti, R. Mantegazza, F. Cornelio

Research output: Contribution to journalArticle

Abstract

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 transplantation is feasible, although the efficacy of this therapeutic approach is poor.

Original languageEnglish
Pages (from-to)291-295
Number of pages5
JournalNeuromuscular Disorders
Volume5
Issue number4
DOIs
Publication statusPublished - 1995

Keywords

  • DMD
  • dystrophin mRNA
  • myoblast transfer

ASJC Scopus subject areas

  • Genetics(clinical)
  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Developmental Neuroscience

Fingerprint Dive into the research topics of 'Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer'. Together they form a unique fingerprint.

  • Cite this