Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia

RNA-targeting approaches are emerging as viable therapeutics that offer an alternative method to modulate traditionally 'undrugable' targets. In the case of dominantly inherited neurodegenerative diseases, gene suppression strategies can target the underlying cause of these intractable dis...

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Veröffentlicht in:	Human molecular genetics 2016-04, Vol.25 (R1), p.R53-R64
Hauptverfasser:	Keiser, Megan S, Kordasiewicz, Holly B, McBride, Jodi L
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Genes, Dominant Humans Huntington Disease - genetics Invited Reviews RNA Interference RNA, Messenger - metabolism Spinocerebellar Ataxias - genetics
Online-Zugang:	Volltext
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