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Article

Antisense oligonucleotide therapy for neurodegenerative disease

28 Jul 2006

Abstract excerpt

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 gene in the CNS. Modified antisense oligonucleotides, continuously infused intraventricularly, have been demonstrated...

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PMID: 16878173
DOI: 10.1172/jci25424

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