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...

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Bibliographic Details
Main Authors: Youxin Gao, Tao Zu, Walter C. Low, Harry T. Orr, R. Scott Mcivor Ph.D.
Format: Article
Language:English
Published: SAGE Publishing 2008-07-01
Series:Cell Transplantation
Online Access:https://doi.org/10.3727/096368908786516729