AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease

MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain

(2017) L Samaranch et al.   GENE THERAPY

The Prevalence of Huntington's Disease

(2016) Michael D. Rawlins et al.   NEUROEPIDEMIOLOGY

In vivo tissue-tropism of adeno-associated viral vectors

(2016) Arun Srivastava   Current Opinion in Virology

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

(2016) Jana Miniarikova et al.   Molecular Therapy-Nucleic Acids

Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo

(2015) Alex Mas Monteys et al.   Molecular Therapy-Nucleic Acids

Huntington disease

(2015) Gillian P. Bates et al.   Nature Reviews Disease Primers

Personalized gene silencing therapeutics for Huntington disease

(2014) C. Kay et al.   CLINICAL GENETICS

Animal models of Huntington's disease for translation to the clinic: Best practices

(2014) Liliana Menalled et al.   MOVEMENT DISORDERS

Huntingtin-lowering strategies in Huntington's disease: Antisense oligonucleotides, small RNAs, and gene editing

(2014) Neil Aronin et al.   MOVEMENT DISORDERS

Huntington disease: natural history, biomarkers and prospects for therapeutics

(2014) Christopher A. Ross et al.   Nature Reviews Neurology

Allele-Specific Silencing of Mutant Huntingtin in Rodent Brain and Human Stem Cells

(2014) Valérie Drouet et al.   PLoS One

Probing the shRNA characteristics that hinder Dicer recognition and consequently allow Ago-mediated processing and AgoshRNA activity

(2014) Elena Herrera-Carrillo et al.   RNA

Designing Ago2-specific siRNA/shRNA to Avoid Competition with Endogenous miRNAs

(2014) Hongming Ma et al.   Molecular Therapy-Nucleic Acids

Choosing an animal model for the study of Huntington's disease

(2013) Mahmoud A. Pouladi et al.   NATURE REVIEWS NEUROSCIENCE

Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

(2012) Richard Grondin et al.   BRAIN

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

(2011) David K. Stiles et al.   EXPERIMENTAL NEUROLOGY

RNAi medicine for the brain: progresses and challenges

(2011) R. L. Boudreau et al.   HUMAN MOLECULAR GENETICS

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

(2011) Jeffrey B Carroll et al.   MOLECULAR THERAPY

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

(2011) Jodi L McBride et al.   MOLECULAR THERAPY

Huntington's disease: from molecular pathogenesis to clinical treatment

(2010) Christopher A Ross et al.   LANCET NEUROLOGY

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

MicroRNAs: Target Recognition and Regulatory Functions

(2009) David P. Bartel   CELL

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

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

A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference

(2009) Maria Stella Lombardi et al.   EXPERIMENTAL NEUROLOGY

Comparative Transduction Efficiency of AAV Vector Serotypes 1–6 in the Substantia Nigra and Striatum of the Primate Brain

(2009) Eleni A Markakis et al.   MOLECULAR THERAPY