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July 30, 2012 / janinezargar

Janine Zargar Discusses a New Treatment for Muscular Dystrophy

A genetic condition, muscular dystrophy causes muscle weakness and a decrease of muscle tissue throughout one’s life. When treating muscular dystrophy and similar disorders, doctors have had difficulty utilizing traditional gene therapy because the mutation is too widespread.

However, a new study shows that forms of muscular dystrophy previously considered untreatable (called dysferlinopathies which are marked by a mutation in the dysferlin gene) may be positively affected by an adeno-associated virus (AAV). While many types of AAVs presented packaging issues due to the size of certain genes, doctors used an AAV serotype known as the AAV5 to work around these limitations. Able to accept larger genes, AAV5s were injected into mice lacking dysferlin through intravascular and intramuscular delivery systems. Results showed full-length, intact dysferlin gene expression in the legs and diaphragms of the subjects.

About the Author:

For nearly 15 years, Janine Zargar has served as Vice President of Doctors United, a New York State Article 28-certified medical facility. Concerned with the welfare of others, Zargar donates to numerous charities including many affiliated with muscular dystrophy research.

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