Breakthrough Against Muscular Dystrophy
Published: April 2, 2006
What’s being called a landmark event could lead to a cure for the most common form of muscular dystrophy, and perhaps other forms of the disease as well.
In Ohio, the first of six boys has undergone the gene therapy that may lead to a cure for Duchenne muscular dystrophy that strikes boys, and usually kills when the patient is in his 20’s.
The boys are missing the muscle protein called dystrophin.
Scientists have figured out a way to insert the gene for dystrophin into a virus. Doctors are injecting the virus into an arm muscle.
The Muscular Dystrophy Association in Tucson made the announcement.
MDA Medical Director Dr. Valerie Cwik says, “The idea is for the virus to basically infect the muscle and insert the new gene, the healthy gene, into the muscle and hope that the muscle will start producing the missing protein,dystrophin.”
This is a safety study, that’s why it’s so small. Dr. Cwik says, if all goes well, “It will be the first of many clinical trials leading to an eventual treatment and perhaps a cure for this disease.”
She says that what scientists learn from this trial will be applicable to other muscular dystrophies.
Dr. Cwik says all this provides a tremendous amount of hope.
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