Antisense oligonucleotide therapy for neurodegenerative disease

Neurotoxicity from accumulation of misfolded/mutant proteins is thought to drive pathogenesis in neurodegenerative diseases. Since decreasing levels of proteins responsible for such accumulations is likely to ameliorate disease, a therapeutic strategy has been developed to downregulate almost any ge...

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Veröffentlicht in:	The Journal of clinical investigation 2006-08, Vol.116 (8), p.2290-2296
Hauptverfasser:	Smith, Richard A, Miller, Timothy M, Yamanaka, Koji, Monia, Brett P, Condon, Thomas P, Hung, Gene, Lobsiger, Christian S, Ward, Chris M, McAlonis-Downes, Melissa, Wei, Hongbing, Wancewicz, Ed V, Bennett, C Frank, Cleveland, Don W
Format:	Artikel
Sprache:	eng
Schlagworte:	Amyotrophic lateral sclerosis Animals Antisense DNA Base Sequence Biomedical research Cerebrospinal fluid Degeneration Disease Fibroblasts - drug effects Fibroblasts - enzymology Macaca mulatta Monkeys & apes Monoclonal antibodies Motor Neuron Disease - enzymology Nervous system Neurodegenerative Diseases - drug therapy Oligonucleotides, Antisense - metabolism Oligonucleotides, Antisense - pharmacology Oligonucleotides, Antisense - therapeutic use Primates Protein Folding Proteins Rats RNA, Messenger - genetics Spinal cord Superoxide Dismutase - genetics Superoxide Dismutase-1 Ventricular Function
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