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- W1585694924 abstract "Neurotrophic factors may be of therapeutic importance for the treatment of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and Parkinson's disease (PD). The transplantation of polymer-encapsulated cells genetically engineered to continuously release neurotrophic factors at a relatively low dose is an extremely useful tool. Cell lines genetically engineered to release the neurotrophic factors CNTF or glial cell line-derived neurotrophic factor (GDNF) have been constructed. The capacity of GDNF to slow down the degeneration of dopaminergic neurons in a rat model of PD has been investigated. The results indicate that the continuous release of low levels of GDNF close to the substantia nigra is capable of protecting nigral dopaminergic neurons from axotomy-induced cell death and improving amphetamine-induced rotation by a mechanism other than dopaminergic striatal reinnervation. A phase I clinical trial using intrathecal delivery of human ciliary neurotrophic factor (CNTF) has been performed in six patients suffering from ALS. The study indicates that significant doses of CNTF can be delivered directly into the central nervous system, and that, in contrast to the side effects associated with systemic delivery of CNTF in ALS patients, no limiting adverse effects are observed in any of the patients exposed to low intrathecal doses of CNTF." @default.
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- W1585694924 date "1997-01-01" @default.
- W1585694924 modified "2023-09-26" @default.
- W1585694924 title "A Gene Therapy Approach for the Treatment of Amyotrophic Lateral Sclerosis and Parkinson's Disease" @default.
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- W1585694924 doi "https://doi.org/10.1016/s1054-3589(08)60899-6" @default.
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