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.