SCIENCE FILE : Science in Brief : New Gene Therapy Method for Muscular Dystrophy Uncovered, but Hurdles Remain

Bone marrow transplants using stem cells can insert a missing protein into muscles in mice with muscular dystrophy, Boston researchers report in today’s Nature. The achievement may open the door to a new way to carry out gene therapy for muscular dystrophy and other diseases, but the risks from the procedure as it now stands are far greater than any potential benefits, according to the researchers.

Molecular biologists Richard Mulligan and Louis Kunkel of Boston’s Children’s Hospital were surprised to find that the transplants introduced the dystrophin gene into muscle tissue. But not enough was introduced to provide therapeutic benefit, they said. The treatment also requires irradiating the mice to destroy their natural bone marrow--a process that is not likely to be attempted in children.

Compiled by Times medical writer Thomas H. Maugh II