Mutant CAG Repeats Effectively Targeted by RNA Interference in SCA7 Cells
Spinocerebellar ataxia type 7 (SCA7) is a human neurodegenerative polyglutamine (polyQ) disease caused by a CAG repeat expansion in the open reading frame of the ATXN7 gene. The allele-selective silencing of mutant transcripts using a repeat-targeting strategy has previously been used for several po...
| Main Authors: | , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2016-12-01
|
| Series: | Genes |
| Subjects: | |
| Online Access: | http://www.mdpi.com/2073-4425/7/12/132 |