Gene Therapy

Paola Leone, Ph.D (Professor of Cell Biology & Director of the Cell and Gene Therapy Center at Rowan School of Osteopathic Medicine) has devoted her entire career to Canavan disease working with patients and in the lab developing treatments. 

In 2000, her original study using gene therapy in Canavan disease was the first time a viral vector was used to treat neurological disease.

With technological advances and a more cell-targeted approach, gene therapy is at a point to make a substantial impact and Canavan is the ideal candidate because it is a disease restricted to the brain and is caused by a single gene mutation.

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“Some Canavan children have mutations that maintain a small amount of enzyme function sufficient enough to support motor function development, but in other children enzyme function is completely lost, thereby preventing any motor function. The ability to increase enzyme function through gene-therapy can promote great improvements in motor development and gives us confidence that achieving global corrective gene expression in targeted cells throughout the brain would prove as an effective treatment and possibly a cure.”

 

— Paola Leone, Ph.D. (Professor of Cell Biology & Director of the Cell and Gene Therapy Center at Rowan School of Osteopathic Medicine)

Experiment