Researchers report progress in muscular dystrophy .
PHILADELPHIA 1996-08-29
University of Pennsylvania researchers on Thursday said a new gene-therapy technique for treating muscular dystrophy disease had shown progress in laboratory animals .
Word of the findings , to be published in the Oct. 1 issue of the journal " Human Gene Therapy , " came in advance of the annual Jerry Lewis Labour Day weekend telethon to raise money for muscular dystrophy research .
Several hurdles must be overcome before the method is used in human trials .
Nevertheless , " a treatment based on the new strategy .... may have the potential to benefit many patients , " the University of Pennsylvania Medical Centre said in a release .
Muscular dystrophy is a fatal illness in which the body 's muscle tissue degenerates and is replaced by fat .
Death strikes in early adulthood .
Individuals with the disease have a non-working version of a gene responsible for producing a crucial muscle protein called dystrophin .
In the study at the University 's Institute for Human Gene Therapy , researchers altered a common-cold virus to carry a version of the working dystrophin gene .
The virus , which also was altered to minimise its susceptibility to the immune system , was then injected into the muscle cells of mice bred to lack dystrophin genes .
In the experiment , between 30 to 40 percent of the muscle fibers in one group of mice produced dystrophin for two weeks before diminishing .
Similar results had been obtained previously in test-tube cell cultures , but not in live animals , the university said .
The university said methods are still needed to make enough of the altered virus to treat humans , to further decrease the immune-system response to the virus , and to deliver the virus to human muscle tissue .