A novel means of drug delivery: myoblast-mediated gene therapy and regulatable retroviral vectors

CR Ozawa, ML Springer, HM Blau - Annual Review of …, 2000 - annualreviews.org
A potentially powerful approach to drug delivery in the treatment of disease involves the use
of cells to introduce genes encoding therapeutic proteins into the body. Candidate genes for
delivery include those encoding secreted factors that could have broad applications ranging
from treatment of inherited single-gene deficiencies to acquired disorders of the vasculature
or cancer. Myoblasts, the proliferative cell type of skeletal muscle tissues, are potent tools for
stable delivery of a gene of interest into the body, as they become an integral part of the …