RESEARCH: Nerve Growth Factor Gene Therapy for Alzheimer's Disease

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http://www.srinstitute.com/cs218

Report from the conference on Neurodegeneration in Alzheimer's, Parkinson's

and Related Disorders

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Nerve Growth Factor Gene Therapy for Alzheimer’s Disease Cholinergic

Neuronal Degeneration

Nerve growth factor (NGF) delivery to the brain can prevent the death of and

reverse the decline of cholinergic neurons in rodent and primate models of

aging and cholinergic neuronal degeneration. Gene therapy, wherein cells

transduced with a therapeutic gene (ex vivo approach) or vectors capable of

infecting non-dividing cells (in vivo approach) are injected into the brain,

offers a feasible approach to overcome problems attaining targeted long-term

delivery of NGF to cholinergic neurons. Ex vivo gene delivery of NGF shows

efficacy in rodent and non-human primate models of cholinergic degeneration

and is currently being investigated in a Phase I clinical trial to treat

Alzheimer’s Disease. In vivo gene delivery of NGF using a viral vector shows

efficacy in the fimbria-fornix lesion model of cholinergic degeneration in

rodents and is currently under further development. These gene therapy

approaches hold promise for delivery of therapeutic genes for the treatment

of neurodegenerative disorders.

Kathie M. Bishop, Ph.D.

Scientist, Alzheimer’s Disease Research

CEREGENE, INC.