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  4. Restoration of cognitive and motor functions by ciliary neurotrophic factor in a primate model of Huntington's disease
 
research article

Restoration of cognitive and motor functions by ciliary neurotrophic factor in a primate model of Huntington's disease

Mittoux, V.
•
Joseph, J. M.
•
Conde, F.
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2000
Human Gene Therapy

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.

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Type
research article
DOI
10.1089/10430340050015220
Author(s)
Mittoux, V.
Joseph, J. M.
Conde, F.
Palfi, S.
Dautry, C.
Poyot, T.
Bloch, J.
Deglon, N.  
Ouary, S.
Nimchinsky, E. A.
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Date Issued

2000

Published in
Human Gene Therapy
Volume

11

Issue

8

Start page

1177

End page

87

Subjects

Animals

•

Brain/metabolism/*pathology

•

Calcium-Binding Protein

•

Vitamin D-Dependent/metabolism

•

Cell Line

•

Ciliary Neurotrophic Factor/administration & dosage/*genetics

•

Convulsants/pharmacology

•

Cricetinae

•

Disease Models

•

Animal

•

Female

•

Gene Therapy/*methods

•

Genetic Vectors

•

Humans

•

Huntington Disease/*therapy

•

Immunohistochemistry

•

Macaca fascicularis

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Magnetic Resonance Imaging

•

Motor Skills

•

Neurobehavioral Manifestations

•

Nitro Compounds

•

Propionic Acids/pharmacology

•

Putamen/metabolism

•

Rats

•

Succinate Dehydrogenase/metabolism

•

Time Factors

•

Transfection

•

Transgenes

Note

URA CEA CNRS 2210, SHFJ, DRM, DSV, CEA, Orsay, France.

Editorial or Peer reviewed

REVIEWED

Written at

EPFL

EPFL units
LEN  
Available on Infoscience
August 27, 2008
Use this identifier to reference this record
https://infoscience.epfl.ch/handle/20.500.14299/27532
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