Growth factor gene therapy for Alzheimer disease.
The capacity to prevent neuronal degeneration and death during the course of progressive neurological disorders such as Alzheimer disease (AD) would represent a significant advance in therapy. Nervous system growth factors are families of naturally produced proteins that, in animal models, exhibit extensive potency in preventing neuronal death due to a variety of causes, reversing age-related atrophy of neurons, and ameliorating functional deficits. The main challenge in translating growth factor therapy to the clinic has been delivery of growth factors to the brain in sufficient concentrations to influence neuronal function. One means of achieving growth factor delivery to the central nervous system in a highly targeted, effective manner may be gene therapy. In this article the authors summarize the development and implementation of nerve growth factor gene delivery as a potential means of reducing cell loss in AD.
Tuszynski
Mark H
MH
Department of Neurosciences, Division of Neurosurgery, University of California at San Diego, La Jolla, California 92093, USA. mtuszynski@ucsd.edu
U
Hoi Sang
HS
Alksne
John
J
Bakay
Roy A
RA
Pay
Mary Margaret
MM
Merrill
David
D
Thal
Leon J
LJ
D000544:Alzheimer Disease / Q000188:drug therapy* / Q000235:genetics*; D000818:Animals; D001921:Brain / Q000187:drug effects; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D020932:Nerve Growth Factor / Q000008:administration & dosage / Q000235:genetics*
D020932:Nerve Growth Factor