Antisense RNA Sequences Modulating the Ataxin-1 Message: Molecular Model of Gene Therapy for Spinocerebellar Ataxia Type 1, a Dominant-Acting Unstable Trinucleotide Repeat Disease
Spinocerebellar ataxia type 1 (SCA1) is a dominant inherited disease caused by expanded trinucleotide repeats resulting in an increased polyglutamine tract in the gene product. As a potential therapeutic approach for SCA1, we tested antisense RNAs targeting two regions of the ataxin-1 message. Singl...
Main Authors: | , , , , |
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Format: | Article |
Language: | English |
Published: |
SAGE Publishing
2008-07-01
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Series: | Cell Transplantation |
Online Access: | https://doi.org/10.3727/096368908786516729 |