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Myoblast implantation in Duchenne muscular dystrophy: The San Francisco study

โœ Scribed by 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

Publisher
John Wiley and Sons
Year
1997
Tongue
English
Weight
300 KB
Volume
20
Category
Article
ISSN
0148-639X

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โœฆ Synopsis

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.

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