Display Settings:

Format

Send to:

Choose Destination
See comment in PubMed Commons below
Mol Ther. 2000 Apr;1(4):304-13.

Immunobiology and the future of myoblast transfer therapy.

Author information

  • 1Department of Anatomy and Human Biology, University of Western Australia, Perth, Australia. gsmythe@anhb.uwa.edu.au

Abstract

Myoblast transfer therapy (MTT) is a cell-mediated gene transfer method aimed at the restoration of normal dystrophin expression in Duchenne muscular dystrophy (DMD). Initial clinical MTT trials were conducted amid much controversy, as they were based on very few animal studies. Unfortunately, the trials were of little therapeutic benefit. As a result, there has been a renaissance of interest in experimental studies in animal models. In MTT, myoblasts are obtained by muscle biopsy from normal, i.e., dystrophin-positive, donors, expanded in culture, and injected directly into the muscles of dystrophic recipients. The major requirement for successful MTT is the survival of injected donor myoblasts in the host environment. However, a vast majority of donor cells fail to survive for more than 1 h after injection, and very few last beyond the first week. This review on the immunological aspects of MTT focuses in particular on the roles of specific components of the host immune response, the effects of tissue culture on donor cells, and strategies under development to circumvent the problem of donor myoblast death after injection in vivo.

PMID:
10933948
[PubMed - indexed for MEDLINE]
Free full text
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Full text links

    Icon for Nature Publishing Group
    Loading ...
    Write to the Help Desk