Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats

Mutant CAG repeats of Huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference

(2011) Mateusz de Mezer et al.   NUCLEIC ACIDS RESEARCH

Partners in crime: bidirectional transcription in unstable microsatellite disease

(2010) R. Batra et al.   HUMAN MOLECULAR GENETICS

Mammalian microRNAs predominantly act to decrease target mRNA levels

(2010) Huili Guo et al.   NATURE

Trinucleotide repeats in human genome and exome

(2010) Piotr Kozlowski et al.   NUCLEIC ACIDS RESEARCH

CAG Expansion in the Huntington Disease Gene Is Associated with a Specific and Targetable Predisposing Haplogroup

(2009) Simon C. Warby et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY

The pathogenic mechanisms of polyglutamine diseases and current therapeutic strategies

(2009) Peter O. Bauer et al.   JOURNAL OF NEUROCHEMISTRY

Asymmetric Shorter-duplex siRNA Structures Trigger Efficient Gene Silencing With Reduced Nonspecific Effects

(2009) Chan Il Chang et al.   MOLECULAR THERAPY

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

(2009) Ryan L Boudreau et al.   MOLECULAR THERAPY

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs

(2009) Jiaxin Hu et al.   NATURE BIOTECHNOLOGY

Profiling of mismatch discrimination in RNAi enabled rational design of allele-specific siRNAs

(2009) Huang Huang et al.   NUCLEIC ACIDS RESEARCH

Antitumor effect of dsRNA-induced p21WAF1/CIP1 gene activation in human bladder cancer cells

(2008) Z. Chen et al.   MOLECULAR CANCER THERAPEUTICS

Asymmetric RNA duplexes mediate RNA interference in mammalian cells

(2008) Xiangao Sun et al.   NATURE BIOTECHNOLOGY

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

(2008) Sandro Alves et al.   PLoS One

Bidirectional Transcription Directs Both Transcriptional Gene Activation and Suppression in Human Cells

(2008) Kevin V. Morris et al.   PLoS Genetics