Search Results - "Huntington's disease"

Biomarkers for Huntington's disease promises and challenges /

Table of Contents: “…Contents: Summary of the genetic, pathophysiology and clinical features of Huntington's disease -- Potential drug targets in development for Huntington's disease -- The importance of biomarkers in Huntington's disease -- Current biomarkers in development in Huntington's disease.…”
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RNAi for neurological diseases

Table of Contents: “…Contents: Repeat expansion diseases -- Polyglutamine (polyQ) repeat diseases -- Huntington's disease -- Vectors for delivering genetic material to brain -- AAVs for delivering RNAi for reducing gene expression -- Artificial miRNAs -- Testing miHD for efficacy -- Off-target silencing -- MRI-guided delivery of AAV into primates -- HTT suppression -- Other gain-of function diseases -- Targeted infusion for broad coverage -- Silencing ataxin-1 -- Nonallele specific silencing: experimental design -- Expression and activity of miSCA1.…”
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Series (RNA interference)
Series (Gene transfer and gene therapy)

Microsatellite and trinucleotide repeat expansion diseases

Table of Contents: “…Contents: Mendelian neurodegenerative diseases are caused by repeat expansions -- The mutation can result in gain-of-function or loss-of-function -- Fragile X syndrome -- Myotonic dystrophy -- Huntington's disease -- Friedrich's ataxia -- C9orf72.…”
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