Restoration of cognitive and motor functions by ciliary neurotrophic factor in a primate model of Huntington's disease.
Details
Serval ID
serval:BIB_1D85A501F855
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Restoration of cognitive and motor functions by ciliary neurotrophic factor in a primate model of Huntington's disease.
Journal
Human Gene Therapy
ISSN
1043-0342 (Print)
ISSN-L
1043-0342
Publication state
Published
Issued date
2000
Peer-reviewed
Oui
Volume
11
Number
8
Pages
1177-1187
Language
english
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't ; Research Support, U.S. Gov't, P.H.S. Publication Status: ppublish
Abstract
Huntington's disease (HD) is an inherited disorder characterized by cognitive impairments, motor deficits, and progressive dementia. These symptoms result from progressive neurodegenerative changes mainly affecting the neostriatum. This pathology is fatal in 10 to 20 years and there is currently no treatment for HD. Early in the course of the disease, initial clinical manifestations are due to striatal neuronal dysfunction, which is later followed by massive neuronal death. A major therapeutic objective is therefore to reverse striatal dysfunction prior to cell death. Using a primate model reproducing the clinical features and the progressive neuronal degeneration typical of HD, we tested the therapeutic effects of direct intrastriatal infusion of ciliary neurotrophic factor (CNTF). To achieve a continuous delivery of CNTF over the full period of evaluation, we took advantage of the macroencapsulation technique. Baby hamster kidney (BHK) cells previously engineered to produce human CNTF were encapsulated into semipermeable membranes and implanted bilaterally into striata. We show here that intracerebral delivery of low doses of CNTF at the onset of symptoms not only protects neurons from degeneration but also restores neostriatal functions. CNTF-treated primates recovered, in particular, cognitive and motor functions dependent on the anatomofunctional integrity of frontostriatal pathways that were distinctively altered in this HD model. These results support the hypothesis that CNTF infusion into the striatum of HD patients not only could block the degeneration of neurons but also alleviated motor and cognitive symptoms associated with persistent neuronal dysfunction.
Keywords
Animals, Brain/metabolism, Brain/pathology, Calbindins, Cell Line, Ciliary Neurotrophic Factor/administration & dosage, Ciliary Neurotrophic Factor/genetics, Convulsants/pharmacology, Cricetinae, Disease Models, Animal, Female, Genetic Therapy/methods, Genetic Vectors, Humans, Huntington Disease/therapy, Immunohistochemistry, Macaca fascicularis, Magnetic Resonance Imaging, Motor Skills, Neurobehavioral Manifestations, Nitro Compounds, Propionates/pharmacology, Putamen/metabolism, Rats, S100 Calcium Binding Protein G/metabolism, Succinate Dehydrogenase/metabolism, Time Factors, Transfection, Transgenes
Pubmed
Web of science
Create date
06/02/2008 10:02
Last modification date
20/08/2019 12:53