Myoblast implantation in Duchenne muscular dystrophy: The San Francisco study

R. G. Miller, K. R. Sharma, G. K. Pavlath, E. Gussoni, M. Mynhier, P. Yu, A. M. Lanctot, C. M. Greco, L. Steinman, H. M. Blau

Research output: Contribution to journalArticle

151 Scopus citations

Abstract

We evaluated myoblast implantation in 10 boys with Duchenne muscular dystrophy (DMD) and absent dystrophin (age 5-10 years) who were implanted with 100 million myoblasts in the anterior tibial muscle of one leg and placebo in the other. Cyclosporine (5 mg/kg/day) was administered for 7 months. Pre- and postimplantation (after 1 and 6 months) muscle biopsies were analyzed. Force generation (tetanic tension and maximum voluntary contraction) was measured monthly in a double-blind design. There was increased force generation in both legs of all boys, probably due to cyclosporine. Using the polymerase chain reaction, evidence of myoblast survival and dystrophin mRNA expression was obtained in 3 patients after 1 month and in 1 patient after 6 months. These studies suggest a salutary effect of cyclosporine upon muscular force generation in Duchenne muscular dystrophy; however, myoblast implantation was not effective in replacing clinically significant amounts of dystrophin in DMD muscle.

Original languageEnglish (US)
Pages (from-to)469-478
Number of pages10
JournalMuscle and Nerve
Volume20
Issue number4
DOIs
StatePublished - Apr 21 1997
Externally publishedYes

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Keywords

  • cell transplantation
  • cyclosporine
  • Duchenne muscular dystrophy
  • gene therapy
  • myoblasts

ASJC Scopus subject areas

  • Clinical Neurology
  • Neuroscience(all)

Cite this

Miller, R. G., Sharma, K. R., Pavlath, G. K., Gussoni, E., Mynhier, M., Yu, P., Lanctot, A. M., Greco, C. M., Steinman, L., & Blau, H. M. (1997). Myoblast implantation in Duchenne muscular dystrophy: The San Francisco study. Muscle and Nerve, 20(4), 469-478. https://doi.org/10.1002/(SICI)1097-4598(199704)20:4<469::AID-MUS10>3.0.CO;2-U