Skip to main content

Table 1 A comparison of oligonucleotide-based strategies for polyQ diseases based on the desired properties of the reagents

From: An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases

PROPERTIES OF REAGENT
STRATEGIES target specificity allele selectivity universality stability in cells delivery in shRNA
Specific silencing by RNAi uncertain no uncertain uncertain yes
Replacement strategy uncertain uncertain uncertain uncertain yes
SNP targeting uncertain yes no uncertain yes
Repeat targeting by siRNA no no yes uncertain yes
Repeat targeting by miRNA uncertain yes yes uncertain yes
Repeat targeting by ASO uncertain yes yes yes no