TABLE 1
FROM:
RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases
E M Denovan-Wright and B L Davidson
BACK TO ARTICLETable 1. RNAi therapy for nucleotide repeat diseases
| Disease | Target transcript | Expansion |
|---|---|---|
| Huntington's disease | Huntingtin | CAG |
| Spinal cerebellar ataxia type 1 (SCA1)a | Ataxin-1 | CAG |
| SCA2 | Ataxin-2 | CAG |
| SCA3 | Ataxin-3 | CAG |
| SCA7 | Ataxin-7 | CAG |
| SCA8 | NAb | CTG |
| DRPLA | Atrophin-1 | CAG |
| Myotonic dystrophy type I (DM1) | Dystrophia myotonica protein kinase (DMPK)c | CUG |
| DM2 | Zinc-finger protein 9d | CCTG |
a This is only a partial listing of the dominant SCAs. For additional information see Schols et al.
b Not assigned.
c Expansion at 3' end of gene; RNA is pathogenic.
d Expansion is in intron 1.
