Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0002736 (amyotrophic lateral sclerosis)
 19,048 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Currently, there are five phase I clinical trials of recombinant adeno-associated viral vectors for the treatment of neurological disorders that are approved or likely to be approved shortly. Two trials are testing different strategies to treat Parkinson's disease (PD), the third trial is aimed at treating Canavan's disease, a pediatric leukodystrophy, the fourth trial targets Alzheimer's disease (AD), and the fifth will attempt to target the lysosomal storage disorder, Batten's disease. All four clinical trials rely on the de novo expression of an enzyme or a trophic factor to correct neuropathology. Ironically, the theories used to choose enzymes for the two PD trials were widely divergent, whereas the enzymatic strategy used for one of the PD trials and the Canavan's trial have remarkable similarities. Other gene therapy treatment strategies for PD and other disorders, such as amyotrophic lateral sclerosis, are also on the horizon.
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PMID:Clinical trials in neurological disorders using AAV vectors: promises and challenges. 1553 49

The collective evidence to date suggests that environmental exposure to excessive amounts of manganese (Mn) can cause a neurodegenerative condition known as manganism. It is now also relatively clear that Mn is involved in the pathogenesis of Alzheimer's disease and at least some prion diseases. The potential involvement of Mn in a panel of other neurodegenerative conditions including Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, and Batten disease has been suggested and investigated, but the results to date are somewhat inconclusive. Herein, previously reported experimental studies investigating the involvement of Mn in the pathogenesis of these conditions are narratively reviewed.
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PMID:Manganese in manganism, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, and Batten disease: A narrative review. 2913 95