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The fate of individual myoblasts after transplantation into muscles of DMD patients

Nature Medicine volume 3, pages 970–977 (1997)Cite this article

Abstract

Muscle biopsies from six patients with Duchenne muscular dystrophy (DMD) participating in a myoblast transplantation clinical trial were reexamined using a fluorescence in situ hybridization (FISH)-based method. Donor nuclei were detected in all biopsies analyzed, including nine where no donor myoblasts were previously thought to be present. In three patients, more than 10% of the original number of donor cells were calculated as present 6 months after implantation. Half of the detected donor nuclei were fused into host myofibers, and of these, nearly 50% produced dystrophin. These findings demonstrate that although donor myoblasts have persisted after injection, their microenvironment influences whether they fuse and express dystrophin. Our methodology could be used for developing new approaches to improve myoblast transfer efficacy and for the analysis of future gene-and/or cell-based therapies of numerous genetic disorders.

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Author information

Authors and Affiliations

  1. Division of Genetics, 300 Longwood Avenue, Boston, Massachusetts, 02115, USA

    Emanuela Gussoni & Louis M. Kunkel

  2. Department of Molecular Pharmacology, Stanford University School of Medicine, Stanford, California, 94305, USA

    Helen M. Blau

  3. The Howard Hughes Medical Institute at Children's Hospital, 300 Longwood Avenue, Boston, Massachusetts, 02115, USA

    Louis M. Kunkel

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  1. Emanuela Gussoni
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  3. Louis M. Kunkel
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Gussoni, E., Blau, H. & Kunkel, L. The fate of individual myoblasts after transplantation into muscles of DMD patients. Nat Med 3, 970–977 (1997). https://doi.org/10.1038/nm0997-970

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