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- Title
ALS, IGF-1 and gene therapy: ‘it's never too late to mend’.
- Authors
Raoul, C.; Aebischer, P.
- Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neuron disease characterized by the selective and progressive loss of motor neurons in the spinal cord, brainstem and cerebral cortex. It typically leads to progressive muscle weakness and neuromuscular respiratory failure. Ciliary neurotrophic factor, brain-derived neurotrophic factor and insulin-like growth factor 1 (IGF-1) have already been evaluated in ALS patients. Disappointingly, systemic delivery of these recombinant proteins did not lead to clinically beneficial effects in ALS patients. It has been reported that a viral vector-mediated delivery of IGF-1 leads to an impressive beneficial effect on the natural history of the disease.
- Subjects
AMYOTROPHIC lateral sclerosis; SOMATOMEDIN; GENE therapy; MOTOR neurons; RECOMBINANT proteins; MUSCLE diseases; THERAPEUTICS
- Publication
Gene Therapy, 2004, Vol 11, Issue 5, p429
- ISSN
0969-7128
- Publication type
Article
- DOI
10.1038/sj.gt.3302204