Nerve Growth Factor Gene Therapy: Activation of Neuronal Responses in Alzheimer Disease.

ABSTRACT:

Importance

Alzheimer disease (AD) is the most common neurodegenerative disorder and lacks effective disease-modifying therapies. In 2001, we initiated a clinical trial of nerve growth factor (NGF) gene therapy in AD, the first effort at gene delivery in an adult neurodegenerative disorder. This program aimed to determine whether a nervous system growth factor prevents or reduces cholinergic neuronal degeneration in patients with AD. We present postmortem findings in 10 patients with survival times ranging from 1 to 10 years after treatment.

Objective

To determine whether degenerating neurons in AD retain an ability to respond to a nervous system growth factor delivered after disease onset.

Design, setting, and participants

Patients in this anatomicopathological study

SUBMITTER: Tuszynski MH 

PROVIDER: S-EPMC4944824 | biostudies-literature | 2015 Oct

REPOSITORIES: biostudies-literature