MYOBLAST IMPLANTATION IN DUCHENNE MUSCULAR-DYSTROPHY - THE SAN-FRANCISCO STUDY

Citation
Rg. Miller et al., MYOBLAST IMPLANTATION IN DUCHENNE MUSCULAR-DYSTROPHY - THE SAN-FRANCISCO STUDY, Muscle & nerve, 20(4), 1997, pp. 469-478
Citations number
45
Language
INGLESE
art.tipo
Article
Categorie Soggetti
Neurosciences
Journal title
Muscle & nerve → ACNP
ISSN journal
0148-639X
Volume
20
Issue
4
Year of publication
1997
Pages
469 - 478
Database
ISI
SICI code
0148-639X(1997)20:4<469:MIIDM->2.0.ZU;2-Z
Abstract
We evaluated myoblast implantation in 10 boys with Duchenne muscular d ystrophy (DMD) and absent dystrophin (age 5-10 years) who were implant ed 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, probab ly 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 generati on in Duchenne muscular dystrophy; however, myoblast implantation was not effective in replacing clinically significant amounts of dystrophi n in DMD muscle. (C) 1997 John Wiley & Sons, Inc.