The Principles and Practice of Myoblast Transfer
Myoblast transfer is a cell transplantation procedure and it consists of injecting suspensions of viable myoblasts into muscle(s) of a living host. Some of the injected myoblasts may ultimately fuse with some of the host muscle fibers and thereby the transferred myoblast nuclei become functional myonuclei of certain muscle fibers of the host. Thus, myoblast transfer can lead to the formation of “mosaic” muscle fibers, in which, in addition to the native myonuclei, the functional myonuclei of the transferred myoblasts are present expressing their genome. This type of myoblast transfer has been used experimentally by several groups of investigators in different rodent species1–10 and the formation of “mosaic” muscle fibers in the host muscle was demonstrated either by the appearance of hybrid forms of strain-specific isoenzymes or by microscopic techniques using nuclear markers. The potential usefulness of myoblast transfer in negating gene defects in some of the host muscle fibers has been raised a few years ago7–11. More recently, transfer of normal allogeneic myoblasts into dystrophin-deficient muscles produced dystrophin-positive fibers that escaped dystrophic damage12,13. In the dy/dy strain of mice, in which the basic genetic and biochemical defect is still unknown, normal myoblast transfer was reported to have negated some of the microscopic pathological features and functional abnormalities of the injected muscles14.On the basisof the successful normal myoblast transfer experments in the mdx mice12,23, invastigation of the feasibility of a similar approsch in the treatment of certain genetic human muscle disease, such as Duchenne muscular dystrophy appeared to be justified.
KeywordsSatellite Cell Duchenne Dystrophy Skeletal Muscle Fiber Dystrophin Gene Fiber Segment
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