Cure Canavan Fund

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These are the Canavan children

waiting for gene therapy…

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These are the children who wait

for your help.

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About

Canavan

They

Experience

Canavan is a rare inherited brain disease.

Children with Canavan do not reach

any developmental milestones

in terms of motor function.

Brain and muscle atrophy
Issues swallowing
Blindness
Spasticity
Seizures

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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 a 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.

“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)

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Help the Canavan children!

100% of proceeds go towards gene therapy