University of Pittsburgh reports best gene delivery to date of protein missing in Duchenne Muscular...( archers because they mistakenly thought...)

University of Pittsburgh reports best gene delivery to date of protein missing in Duchenne Muscular Dystrophy

archers because they mistakenly thought these tiny microbes failed to infect non-dividing cells and because they could only carry a small genetic payload. Yet, scientists are now quite excited by the biological properties of AAV, according to Dr. Xiao.

The AAV vector is considered ideal for clinical gene therapy for many reasons, according to Dr. Xiao. The virus from which the AAV vector is produced does not cause any diseases and is disabled so that it cannot reproduce. The AAV vector is easily manufactured and stable. It can be targeted to specific tissues and produces long-lasting gene expression. Moreover, it does not provoke an immune response against cells that it enters. This type of immune response has been seen in gene therapies using other virus types.

In a next step, Dr. Xiaos laboratory will collaborate with other investigators in testing the AAV mini-gene construct in a dog model of DMD. Other research initiatives include developing the best ways at delivering the DMD mini-gene systemically, as opposed to performing localized injections.

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Contact: Lauren Ward
wardle@msx.upmc.edu
412-624-2607
University of Pittsburgh Medical Center
27-Nov-2000

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