Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia

Nanoparticle Delivered VEGF-A siRNA Enhances Photodynamic Therapy for Head and Neck Cancer Treatment

(2016) Rumwald Leo G Lecaros et al.   MOLECULAR THERAPY

Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy

(2015) Megan S. Keiser et al.   BRAIN

Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington’s disease patients

(2015) Edward J. Wild et al.   JOURNAL OF CLINICAL INVESTIGATION

A SNP in the HTT promoter alters NF-κB binding and is a bidirectional genetic modifier of Huntington disease

(2015) Kristina Bečanović et al.   NATURE NEUROSCIENCE

Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression

(2015) Amber L. Southwell et al.   Scientific Reports

Lentiviral vectors as tools to understand central nervous system biology in mammalian model organisms

(2015) Louise C. Parr-Brownlie et al.   Frontiers in Molecular Neuroscience

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

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

Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA

(2015) Richard Grondin et al.   Molecular Therapy-Nucleic Acids

Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease

(2014) Lisa M. Stanek et al.   HUMAN GENE THERAPY

Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy

(2014) Megan S Keiser et al.   MOLECULAR THERAPY

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7

(2014) Pavitra S Ramachandran et al.   MOLECULAR THERAPY

Intrajugular Vein Delivery of AAV9-RNAi Prevents Neuropathological Changes and Weight Loss in Huntington's Disease Mice

(2014) Brett D Dufour et al.   MOLECULAR THERAPY

Regulation of microRNA biogenesis

(2014) Minju Ha et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

RNA Interference-Based Therapy for Spinocerebellar Ataxia Type 7 Retinal Degeneration

(2014) Pavitra S. Ramachandran et al.   PLoS One

Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

(2014) Niels H. Skotte et al.   PLoS One

Biology of adeno-associated viral vectors in the central nervous system

(2014) Giridhar Murlidharan et al.   Frontiers in Molecular Neuroscience

Characterization of depression in prodromal Huntington disease in the neurobiological predictors of HD (PREDICT-HD) study

(2013) Eric A. Epping et al.   JOURNAL OF PSYCHIATRIC RESEARCH

Cognitive Reserve and Brain Reserve in Prodromal Huntington's Disease

(2013) Aaron Bonner-Jackson et al.   JOURNAL OF THE INTERNATIONAL NEUROPSYCHOLOGICAL SOCIETY

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Toward RNAi Therapy for the Polyglutamine Disease Machado–Joseph Disease

(2013) Maria do Carmo Costa et al.   MOLECULAR THERAPY

Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice

(2013) Edgardo Rodríguez-Lebrón et al.   MOLECULAR THERAPY

RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1

(2013) Megan S. Keiser et al.   NEUROBIOLOGY OF DISEASE

Recent Advances in RNA Interference Therapeutics for CNS Diseases

(2013) Pavitra S. Ramachandran et al.   Neurotherapeutics

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

(2013) Michael E. Østergaard et al.   NUCLEIC ACIDS RESEARCH

Silencing Mutant Ataxin-3 Rescues Motor Deficits and Neuropathology in Machado-Joseph Disease Transgenic Mice

(2013) Clévio Nóbrega et al.   PLoS One

Clinical features, neurogenetics and neuropathology of the polyglutamine spinocerebellar ataxias type 1, 2, 3, 6 and 7

(2013) Udo Rüb et al.   PROGRESS IN NEUROBIOLOGY

Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

(2013) Vedell Louis Jeune et al.   Human Gene Therapy Methods

Neuroanatomical correlates of cognitive functioning in prodromal Huntington disease

(2013) Deborah L. Harrington et al.   Brain and Behavior

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

Single-Stranded siRNAs Activate RNAi in Animals

(2012) Walt F. Lima et al.   CELL

The advent of AAV9 expands applications for brain and spinal cord gene delivery

(2012) Robert D Dayton et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Cognitive domains that predict time to diagnosis in prodromal Huntington disease

(2012) Deborah Lynn Harrington et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

“Huntingtin Holiday”: Progress toward an Antisense Therapy for Huntington's Disease

(2012) Xiao-Hong Lu et al.   NEURON

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice

(2012) M. Garriga-Canut et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

Mutant Huntingtin Causes Metabolic Imbalance by Disruption of Hypothalamic Neurocircuits

(2011) Sofia Hult et al.   Cell Metabolism

Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

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

Rational Design of Therapeutic siRNAs: Minimizing Off-targeting Potential to Improve the Safety of RNAi Therapy for Huntington's Disease

(2011) Ryan L Boudreau et al.   MOLECULAR THERAPY

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

Neutralizing Antibodies Against AAV Serotypes 1, 2, 6, and 9 in Sera of Commonly Used Animal Models

(2011) Kleopatra Rapti et al.   MOLECULAR THERAPY

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Allele-Selective Inhibition of MutantHuntingtinExpression with Antisense Oligonucleotides Targeting the Expanded CAG Repeat

(2010) Keith T. Gagnon et al.   BIOCHEMISTRY

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Silencing ataxin-3 mitigates degeneration in a rat model of Machado–Joseph disease: no role for wild-type ataxin-3?

(2010) Sandro Alves et al.   HUMAN MOLECULAR GENETICS

A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy

(2010) Natalia N. Singh et al.   RNA Biology

Sustained effects of nonallele-specificHuntingtinsilencing

(2009) Valérie Drouet et al.   ANNALS OF NEUROLOGY

Allele-Selective Inhibition of Mutant Huntingtin by Peptide Nucleic Acid-Peptide Conjugates, Locked Nucleic Acid, and Small Interfering RNA

(2009) Jiaxin Hu et al.   Annals of the New York Academy of Sciences

Cellular localization and allele-selective inhibition of mutant huntingtin protein by peptide nucleic acid oligomers containing the fluorescent nucleobase [bis-o-(aminoethoxy)phenyl]pyrrolocytosine

(2009) Jiaxin Hu et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

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

Ribonuclease H: the enzymes in eukaryotes

(2009) Susana M. Cerritelli et al.   FEBS Journal

Biological and clinical manifestations of Huntington's disease in the longitudinal TRACK-HD study: cross-sectional analysis of baseline data

(2009) Sarah J Tabrizi et al.   LANCET NEUROLOGY

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

Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice

(2008) Yimin Hua et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Molecular pathogenesis and cellular pathology of spinocerebellar ataxia type 7 neurodegeneration

(2008) Gwenn A. Garden et al.   CEREBELLUM

Identification and Allele-Specific Silencing of the Mutant Huntingtin Allele in Huntington's Disease Patient-Derived Fibroblasts

(2008) P.H.J. van Bilsen et al.   HUMAN GENE THERAPY

Delivery Vehicles for Small Interfering RNA In Vivo

(2008) Antonin R. de Fougerolles   HUMAN GENE THERAPY

AAV Vector–mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease

(2008) Nicholas R Franich et al.   MOLECULAR THERAPY

Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo

(2008) Ryan L Boudreau et al.   MOLECULAR THERAPY

Detection of Intact rAAV Particles up to 6 Years After Successful Gene Transfer in the Retina of Dogs and Primates

(2008) Knut Stieger et al.   MOLECULAR THERAPY

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

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

(2008) J. L. McBride et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA