Rapid death of injected myoblasts in myoblast transfer therapy

Y Fan, M Maley, M Beilharz… - Muscle & Nerve: Official …, 1996 - Wiley Online Library
Y Fan, M Maley, M Beilharz, M Grounds
Muscle & Nerve: Official Journal of the American Association of …, 1996Wiley Online Library
Myoblast transplantation has been proposed as a potential therapy for Duchenne muscular
dystrophy (DMD). AY‐chromosome‐specific probe was used to track the fate of donor male
myoblasts injected into dystrophic muscles of female mdx mice (which are an animal model
for DMD). In situ analysis with the Y‐probe showed extremely poor survival of isolated
normal male (C57B1/10Sn) donor myoblasts after injection into injured or uninjured muscles
of dystrophic (mdx) and normal (C57B1/10Sn) female host mice. A decrease in the numbers …
Abstract
Myoblast transplantation has been proposed as a potential therapy for Duchenne muscular dystrophy (DMD). A Y‐chromosome‐specific probe was used to track the fate of donor male myoblasts injected into dystrophic muscles of female mdx mice (which are an animal model for DMD). In situ analysis with the Y‐probe showed extremely poor survival of isolated normal male (C57B1/10Sn) donor myoblasts after injection into injured or uninjured muscles of dystrophic (mdx) and normal (C57B1/10Sn) female host mice. A decrease in the numbers of donor (male) myoblasts was seen from 2 days and was marked by 7 days after injection: few or no donor myoblasts were detected in host muscles examined at 3–12 months. There was limited movement of the injected donor myoblasts and fusion into host myofibers was rare. The results of this study strongly suggest that the failure of clinical trials of myoblast transplantation therapy in boys with DMD may have been due to rapid and massive death of the donor myoblasts soon after myoblast injection. © 1996 John Wiley & Sons, Inc.
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