标题
Therapeutic antisense oligonucleotides for movement disorders
作者
关键词
-
出版物
MEDICINAL RESEARCH REVIEWS
Volume -, Issue -, Pages -
出版商
Wiley
发表日期
2020-07-13
DOI
10.1002/med.21706
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Systemic Brain Delivery of Antisense Oligonucleotides across the Blood-Brain Barrier with a Glucose-Coated Polymeric Nanocarrier
- (2020) Hyun Su Min et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- Targeting Huntingtin Expression in Patients with Huntington’s Disease
- (2019) Sarah J. Tabrizi et al. NEW ENGLAND JOURNAL OF MEDICINE
- RVG-modified exosomes derived from mesenchymal stem cells rescue memory deficits by regulating inflammatory responses in a mouse model of Alzheimer’s disease
- (2019) Guo-hong Cui et al. Immunity & Ageing
- Antisense Oligonucleotide Therapies for Neurodegenerative Diseases
- (2019) C. Frank Bennett et al. Annual Review of Neuroscience
- RNases H: Structure and mechanism
- (2019) Malwina Hyjek et al. DNA REPAIR
- Amido-bridged nucleic acid (AmNA)-modified antisense oligonucleotides targeting α-synuclein as a novel therapy for Parkinson’s disease
- (2019) Takuya Uehara et al. Scientific Reports
- Molecular Targets and Therapeutic Strategies in Spinocerebellar Ataxia Type 7
- (2019) Anna Niewiadomska-Cimicka et al. Neurotherapeutics
- Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide
- (2019) Eleni Kourkouta et al. Molecular Therapy-Nucleic Acids
- Evaluation of off‐target effects of gapmer antisense oligonucleotides using human cells
- (2019) Tokuyuki Yoshida et al. GENES TO CELLS
- Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
- (2019) Jinkuk Kim et al. NEW ENGLAND JOURNAL OF MEDICINE
- Identification of novel risk loci, causal insights, and heritable risk for Parkinson's disease: a meta-analysis of genome-wide association studies
- (2019) Mike A Nalls et al. LANCET NEUROLOGY
- LRRK2 Biology from structure to dysfunction: research progresses, but the themes remain the same
- (2019) Daniel C. Berwick et al. Molecular Neurodegeneration
- Neurochemical abnormalities in premanifest and early spinocerebellar ataxias
- (2018) James M. Joers et al. ANNALS OF NEUROLOGY
- Oligonucleotide therapy mitigates disease in Spinocerebellar Ataxia Type 3 mice
- (2018) Hayley S. McLoughlin et al. ANNALS OF NEUROLOGY
- Cholesterol-Modified Caged siRNAs for Photoregulating Exogenous and Endogenous Gene Expression
- (2018) Jiali Yang et al. BIOCONJUGATE CHEMISTRY
- RNA-Targeted Therapeutics
- (2018) Stanley T. Crooke et al. Cell Metabolism
- Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models
- (2018) Alex McCampbell et al. JOURNAL OF CLINICAL INVESTIGATION
- Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
- (2018) Silvana M.G. Jirka et al. MOLECULAR THERAPY
- Selective α-Synuclein Knockdown in Monoamine Neurons by Intranasal Oligonucleotide Delivery: Potential Therapy for Parkinson’s Disease
- (2018) Diana Alarcón-Arís et al. MOLECULAR THERAPY
- Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic
- (2018) S. Pablo Sardi et al. MOVEMENT DISORDERS
- Severe persistent injection site reactions after subcutaneous 2′-O-methyl phosphorothioate oligonucleotide therapy for Duchenne muscular dystrophy
- (2018) Joana Domingos et al. NEUROMUSCULAR DISORDERS
- Reversible Conformational Conversion of α-Synuclein into Toxic Assemblies by Glucosylceramide
- (2018) Friederike Zunke et al. NEURON
- Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
- (2018) Eugenio Mercuri et al. NEW ENGLAND JOURNAL OF MEDICINE
- Neuropathology of Parkinson disease
- (2018) Dennis W. Dickson PARKINSONISM & RELATED DISORDERS
- GBA1 deficiency negatively affects physiological α-synuclein tetramers and related multimers
- (2018) Sangjune Kim et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- α-synuclein oligomers interact with ATP synthase and open the permeability transition pore in Parkinson’s disease
- (2018) Marthe H. R. Ludtmann et al. Nature Communications
- Chronic Toxicity Assessment of 2′-O-Methoxyethyl Antisense Oligonucleotides in Mice
- (2018) Thomas A. Zanardi et al. Nucleic Acid Therapeutics
- Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver
- (2018) Colton M. Miller et al. Nucleic Acid Therapeutics
- Progression in the LRRK2-Associated Parkinson Disease Population
- (2018) Rachel Saunders-Pullman et al. JAMA Neurology
- A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides
- (2018) Andreas Dieckmann et al. Molecular Therapy-Nucleic Acids
- Exosome-based small RNA delivery: Progress and prospects
- (2018) Mei Lu et al. Asian Journal of Pharmaceutical Sciences
- Nuclear localization and phosphorylation modulate pathological effects of Alpha-Synuclein
- (2018) Raquel Pinho et al. HUMAN MOLECULAR GENETICS
- Central and Peripheral Administration of Antisense Oligonucleotide Targeting Amyloid-β Protein Precursor Improves Learning and Memory and Reduces Neuroinflammatory Cytokines in Tg2576 (AβPPswe) Mice
- (2018) Susan A. Farr et al. JOURNAL OF ALZHEIMERS DISEASE
- Biological and clinical manifestations of juvenile Huntington's disease: a retrospective analysis
- (2018) Caterina Fusilli et al. LANCET NEUROLOGY
- Loss of the Spinocerebellar Ataxia type 3 disease protein ATXN3 alters transcription of multiple signal transduction pathways
- (2018) Li Zeng et al. PLoS One
- Evaluation of the effect of 2′-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides in mice
- (2018) Thazha P. Prakash et al. BIOORGANIC & MEDICINAL CHEMISTRY LETTERS
- Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study
- (2018) Niels Hilhorst et al. EUROPEAN JOURNAL OF PEDIATRICS
- A microRNA-29 mimic (remlarsen) represses extracellular matrix expression and fibroplasia in the skin
- (2018) Corrie L. Gallant-Behm et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Heterozygous STUB1 mutation causes familial ataxia with cognitive affective syndrome (SCA48)
- (2018) David Genis et al. NEUROLOGY
- Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease
- (2018) Amber L. Southwell et al. Science Translational Medicine
- Molecular pathway analysis towards understanding tissue vulnerability in spinocerebellar ataxia type 1
- (2018) Terri M Driessen et al. eLife
- Huntington's disease: a clinical review
- (2017) P. McColgan et al. EUROPEAN JOURNAL OF NEUROLOGY
- Lipid-oligonucleotide conjugates improve cellular uptake and efficiency of TCTP-antisense in castration-resistant prostate cancer
- (2017) Sara Karaki et al. JOURNAL OF CONTROLLED RELEASE
- Safety, tolerability, and antiviral effect of RG-101 in patients with chronic hepatitis C: a phase 1B, double-blind, randomised controlled trial
- (2017) Meike H van der Ree et al. LANCET
- Antisense oligonucleotide therapy for spinocerebellar ataxia type 2
- (2017) Daniel R. Scoles et al. NATURE
- Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides
- (2017) Naoki Iwamoto et al. NATURE BIOTECHNOLOGY
- Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
- (2017) Richard S. Finkel et al. NEW ENGLAND JOURNAL OF MEDICINE
- Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes
- (2017) Michael Tanowitz et al. NUCLEIC ACIDS RESEARCH
- Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor
- (2017) Karsten Schmidt et al. NUCLEIC ACIDS RESEARCH
- Nucleic acid binding proteins affect the subcellular distribution of phosphorothioate antisense oligonucleotides
- (2017) Jeffrey K. Bailey et al. NUCLEIC ACIDS RESEARCH
- Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy
- (2017) Sarah L. DeVos et al. Science Translational Medicine
- Gene Silencing Activity and Hepatic Accumulation of Antisense Oligonucleotides Bearing Cholesterol-Conjugated Thiono Triester at the Gap Region
- (2017) Mado Nakajima et al. Nucleic Acid Therapeutics
- The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials
- (2017) Stanley T. Crooke et al. Nucleic Acid Therapeutics
- The Role of Lipids Interacting with α-Synuclein in the Pathogenesis of Parkinson’s Disease
- (2017) Céline Galvagnion Journal of Parkinsons Disease
- Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis
- (2017) Andrew P. Tosolini et al. Frontiers in Molecular Neuroscience
- Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice
- (2017) Lodewijk J.A. Toonen et al. Molecular Therapy-Nucleic Acids
- Cholesterol-Containing Nuclease-Resistant siRNA Accumulates in Tumors in a Carrier-free Mode and Silences MDR1 Gene
- (2017) Ivan V. Chernikov et al. Molecular Therapy-Nucleic Acids
- LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model
- (2017) Hien Tran Zhao et al. Molecular Therapy-Nucleic Acids
- Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models
- (2017) Lauren R. Moore et al. Molecular Therapy-Nucleic Acids
- The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain
- (2017) Nicole A. Datson et al. PLoS One
- Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides
- (2016) Elizabeth J. Ackermann et al. AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- Evaluation of anhydrohexitol nucleic acid, cyclohexenyl nucleic acid and d-altritol nucleic acid-modified 2′-O-methyl RNA mixmer antisense oligonucleotides for exon skipping in vitro
- (2016) Bao T. Le et al. CHEMICAL COMMUNICATIONS
- Antisense targeting of 3′ end elements involved inDUX4mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach
- (2016) Anne-Charlotte Marsollier et al. HUMAN MOLECULAR GENETICS
- Targeting CAG repeat RNAs reduces Huntington’s disease phenotype independently of huntingtin levels
- (2016) Laura Rué et al. JOURNAL OF CLINICAL INVESTIGATION
- The clinical symptoms of Parkinson's disease
- (2016) Sigurlaug Sveinbjornsdottir JOURNAL OF NEUROCHEMISTRY
- G2019S-LRRK2 Expression Augments -Synuclein Sequestration into Inclusions in Neurons
- (2016) L. A. Volpicelli-Daley et al. JOURNAL OF NEUROSCIENCE
- Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing
- (2016) Marie-Cécile Didiot et al. MOLECULAR THERAPY
- Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics
- (2016) Jennifer CJ Chen et al. MOLECULAR THERAPY
- Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line
- (2016) James J. Dowling Nature Reviews Neurology
- Next-generation sequencing reveals substantial genetic contribution to dementia with Lewy bodies
- (2016) Joshua T. Geiger et al. NEUROBIOLOGY OF DISEASE
- The Prevalence of Huntington's Disease
- (2016) Michael D. Rawlins et al. NEUROEPIDEMIOLOGY
- The Biology of Huntingtin
- (2016) Frédéric Saudou et al. NEURON
- Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy
- (2016) Suzan M. Hammond et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis
- (2016) Guohao Wang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3
- (2016) Lodewijk J. A. Toonen et al. Scientific Reports
- Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study
- (2016) Nathalie M. Goemans et al. PLoS One
- Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides
- (2016) Takeshi Kasuya et al. Scientific Reports
- Miravirsen dosing in chronic hepatitis C patients results in decreased microRNA-122 levels without affecting other microRNAs in plasma
- (2015) M. H. van der Ree et al. ALIMENTARY PHARMACOLOGY & THERAPEUTICS
- An orally administrated nucleotide-delivery vehicle targeting colonic macrophages for the treatment of inflammatory bowel disease
- (2015) Zhen Huang et al. BIOMATERIALS
- Phosphorothioate backbone modifications of nucleotide-based drugs are potent platelet activators
- (2015) Ulrike Flierl et al. JOURNAL OF EXPERIMENTAL MEDICINE
- Long-term disease progression in spinocerebellar ataxia types 1, 2, 3, and 6: a longitudinal cohort study
- (2015) Heike Jacobi et al. LANCET NEUROLOGY
- Assessing recovery from neurodegeneration in spinocerebellar ataxia 1: Comparison of in vivo magnetic resonance spectroscopy with motor testing, gene expression and histology
- (2015) Gülin Öz et al. NEUROBIOLOGY OF DISEASE
- Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts
- (2015) Sebastien A. Burel et al. NUCLEIC ACIDS RESEARCH
- 2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF
- (2015) Wen Shen et al. NUCLEIC ACIDS RESEARCH
- Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
- (2015) Ole B Suhr et al. Orphanet Journal of Rare Diseases
- Allele-Selective Inhibition of Mutant Huntingtin with 2-Thio- and C5- Triazolylphenyl-Deoxythymidine-Modified Antisense Oligonucleotides
- (2015) Michael E. Østergaard et al. Nucleic Acid Therapeutics
- Usefulness of Genetic Testing in PD and PD Trials: A Balanced Review
- (2015) Thomas Gasser Journal of Parkinsons Disease
- Ataxin-2 Regulates RGS8 Translation in a New BAC-SCA2 Transgenic Mouse Model
- (2015) Warunee Dansithong et al. PLoS Genetics
- Population Genetics and New Insight into Range of CAG Repeats of Spinocerebellar Ataxia Type 3 in the Han Chinese Population
- (2015) Shi-Rui Gan et al. PLoS One
- Systemic Delivery of a Peptide-Linked Morpholino Oligonucleotide Neutralizes Mutant RNA Toxicity in a Mouse Model of Myotonic Dystrophy
- (2015) Andrew J. Leger et al. Nucleic Acid Therapeutics
- MicroRNA mimicry blocks pulmonary fibrosis
- (2014) R. L. Montgomery et al. EMBO Molecular Medicine
- Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity
- (2014) Xin Sun et al. HUMAN MOLECULAR GENETICS
- 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
- Ten Years and Counting: Moving Leucine-Rich RepeatKinase 2 Inhibitors to the Clinic
- (2014) Andrew B. West MOVEMENT DISORDERS
- The Global Epidemiology of Hereditary Ataxia and Spastic Paraplegia: A Systematic Review of Prevalence Studies
- (2014) Luis Ruano et al. NEUROEPIDEMIOLOGY
- Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides
- (2014) Amanda J. Ward et al. NUCLEIC ACIDS RESEARCH
- Phosphorothioate oligonucleotides can displaceNEAT1RNA and form nuclear paraspeckle-like structures
- (2014) Wen Shen et al. NUCLEIC ACIDS RESEARCH
- Phosphorothioates, Essential Components of Therapeutic Oligonucleotides
- (2014) Fritz Eckstein Nucleic Acid Therapeutics
- Mechanism of Alternative Complement Pathway Dysregulation by a Phosphorothioate Oligonucleotide in Monkey and Human Serum
- (2014) Scott P. Henry et al. Nucleic Acid Therapeutics
- Mipomersen, an Apolipoprotein B Synthesis Inhibitor, Reduces Atherogenic Lipoproteins in Patients With Severe Hypercholesterolemia at High Cardiovascular Risk
- (2013) Gregory S. Thomas et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- rAAV2/7 vector-mediated overexpression of alpha-synuclein in mouse substantia nigra induces protein aggregation and progressive dose-dependent neurodegeneration
- (2013) Marusela Oliveras-Salvá et al. Molecular Neurodegeneration
- Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon
- (2013) Melvin M. Evers et al. NEUROBIOLOGY OF DISEASE
- Miravirsen (SPC3649) can inhibit the biogenesis of miR-122
- (2013) Luca F. R. Gebert et al. NUCLEIC ACIDS RESEARCH
- Clinical characteristics of patients with spinocerebellar ataxias 1, 2, 3 and 6 in the US; a prospective observational study
- (2013) Tetsuo Ashizawa et al. Orphanet Journal of Rare Diseases
- Hepatotoxic Potential of Therapeutic Oligonucleotides Can Be Predicted from Their Sequence and Modification Pattern
- (2013) Peter H. Hagedorn et al. Nucleic Acid Therapeutics
- Peptide Conjugation of 2′-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice
- (2013) Silvana M.G. Jirka et al. Nucleic Acid Therapeutics
- The EPR effect for macromolecular drug delivery to solid tumors: Improvement of tumor uptake, lowering of systemic toxicity, and distinct tumor imaging in vivo
- (2012) Hiroshi Maeda et al. ADVANCED DRUG DELIVERY REVIEWS
- The scavenger endothelial cell: a new player in homeostasis and immunity
- (2012) Karen Kristine Sørensen et al. AMERICAN JOURNAL OF PHYSIOLOGY-REGULATORY INTEGRATIVE AND COMPARATIVE PHYSIOLOGY
- Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B
- (2012) Stanley T. Crooke et al. BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
- Spinocerebellar Ataxia Type 7: Clinical Course, Phenotype–Genotype Correlations, and Neuropathology
- (2012) Laura C. Horton et al. CEREBELLUM
- Selective binding of nuclear alpha-synuclein to the PGC1alpha promoter under conditions of oxidative stress may contribute to losses in mitochondrial function: Implications for Parkinson’s disease
- (2012) Almas Siddiqui et al. FREE RADICAL BIOLOGY AND MEDICINE
- Current understanding of LRRK2 in Parkinson’s disease: biochemical and structural features and inhibitor design
- (2012) Soumya Ray et al. Future Medicinal Chemistry
- Changes in Purkinje cell firing and gene expression precede behavioral pathology in a mouse model of SCA2
- (2012) S. T. Hansen et al. HUMAN MOLECULAR GENETICS
- Spinocerebellar Ataxia Type 2: Clinical Presentation, Molecular Mechanisms, and Therapeutic Perspectives
- (2012) J. J. Magaña et al. MOLECULAR NEUROBIOLOGY
- Exosome-mediated delivery of siRNA in vitro and in vivo
- (2012) Samir El-Andaloussi et al. Nature Protocols
- Penetrance of Parkinson disease in glucocerebrosidase gene mutation carriers
- (2012) M. Anheim et al. NEUROLOGY
- Cognitive performance of GBA mutation carriers with early-onset PD: The CORE-PD study
- (2012) R. N. Alcalay et al. NEUROLOGY
- Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
- (2012) Holly B. Kordasiewicz et al. NEURON
- Quantitative Assessment of Brain Stem and Cerebellar Atrophy in Spinocerebellar Ataxia Types 3 and 6: Impact on Clinical Status
- (2011) L. Eichler et al. AMERICAN JOURNAL OF NEURORADIOLOGY
- Ataxin-1 and Brother of ataxin-1 are components of the Notch signalling pathway
- (2011) Xin Tong et al. EMBO REPORTS
- In vivo monitoring of recovery from neurodegeneration in conditional transgenic SCA1 mice
- (2011) Gülin Öz et al. EXPERIMENTAL NEUROLOGY
- Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum
- (2011) Liutao Du et al. HUMAN MOLECULAR GENETICS
- Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
- (2011) Sebahattin Cirak et al. LANCET
- The novel antisense Bcl-2 inhibitor SPC2996 causes rapid leukemic cell clearance and immune activation in chronic lymphocytic leukemia
- (2011) J Dürig et al. LEUKEMIA
- 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
- Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes
- (2011) Lydia Alvarez-Erviti et al. NATURE BIOTECHNOLOGY
- The Machado-Joseph disease deubiquitylase ATX-3 couples longevity and proteostasis
- (2011) Kirsten Kuhlbrodt et al. NATURE CELL BIOLOGY
- Carrier-free cellular uptake and the gene-silencing activity of the lipophilic siRNAs is strongly affected by the length of the linker between siRNA and lipophilic group
- (2011) Natalya S. Petrova et al. NUCLEIC ACIDS RESEARCH
- Machado-Joseph Disease: from first descriptions to new perspectives
- (2011) Conceição Bettencourt et al. Orphanet Journal of Rare Diseases
- Toward understanding Machado–Joseph disease
- (2011) Maria do Carmo Costa et al. PROGRESS IN NEUROBIOLOGY
- Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
- (2011) M. A. Passini et al. Science Translational Medicine
- Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
- (2010) Y. Hua et al. GENES & DEVELOPMENT
- Transcriptional changes in Huntington disease identified using genome-wide expression profiling and cross-platform analysis
- (2010) K. Becanovic et al. HUMAN MOLECULAR GENETICS
- Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial
- (2010) Frederick J Raal et al. LANCET
- Biological and clinical changes in premanifest and early stage Huntington's disease in the TRACK-HD study: the 12-month longitudinal analysis
- (2010) Sarah J Tabrizi et al. LANCET NEUROLOGY
- PolyQ Disease: Too Many Qs, Too Much Function?
- (2010) Ian H. Kratter et al. NEURON
- -Synuclein Promotes SNARE-Complex Assembly in Vivo and in Vitro
- (2010) J. Burre et al. SCIENCE
- Infliximab and other immunomodulating drugs in patients with inflammatory bowel disease and the risk of serious bacterial infections
- (2009) S. SCHNEEWEISS et al. ALIMENTARY PHARMACOLOGY & THERAPEUTICS
- Antisense oligonucleotide pharmacokinetics and metabolism
- (2009) Richard S Geary Expert Opinion on Drug Metabolism & Toxicology
- Dystrophin restoration in skeletal, heart and skin arrector pili smooth muscle of mdx mice by ZM2 NP–AON complexes
- (2009) A Ferlini et al. GENE THERAPY
- Cellular uptake and biological activity of peptide nucleic acids conjugated with peptides with and without cell-penetrating ability
- (2009) Yvonne Turner et al. JOURNAL OF PEPTIDE SCIENCE
- Cationic PMMA Nanoparticles Bind and Deliver Antisense Oligoribonucleotides Allowing Restoration of Dystrophin Expression in the mdx Mouse
- (2009) Paola Rimessi 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
- GLUCOCEREBROSIDASE MUTATIONS IN 108 NEUROPATHOLOGICALLY CONFIRMED CASES OF MULTIPLE SYSTEM ATROPHY
- (2009) B. Segarane et al. NEUROLOGY
- Nucleocytoplasmic Shuttling Activity of Ataxin-3
- (2009) Sandra Macedo-Ribeiro et al. PLoS One
- Dissociated Fear and Spatial Learning in Mice with Deficiency of Ataxin-2
- (2009) Duong P. Huynh et al. PLoS One
- Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA
- (2009) T. M. Wheeler et al. SCIENCE
- Caveolae and transcytosis in endothelial cells: role in atherosclerosis
- (2008) Philippe G. Frank et al. CELL AND TISSUE RESEARCH
- SCA3: Neurological features, pathogenesis and animal models
- (2008) Olaf Riess et al. CEREBELLUM
- Transferrin-conjugated liposomal system for improved delivery of 5-fluorouracil to brain
- (2008) Vandana Soni et al. JOURNAL OF DRUG TARGETING
- Phenotype, genotype, and worldwide genetic penetrance of LRRK2-associated Parkinson's disease: a case-control study
- (2008) Daniel G Healy et al. LANCET NEUROLOGY
- New therapeutic approach for brain tumors: Intranasal delivery of telomerase inhibitor GRN163
- (2008) Rintaro Hashizume et al. NEURO-ONCOLOGY
- Polyglutamine-expanded ataxin-3 causes cerebellar dysfunction of SCA3 transgenic mice by inducing transcriptional dysregulation
- (2008) An-Hsun Chou et al. NEUROBIOLOGY OF DISEASE
- Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle
- (2008) Gabriela D. Ivanova et al. NUCLEIC ACIDS RESEARCH
- Properties of the Glomerular Barrier and Mechanisms of Proteinuria
- (2008) Börje Haraldsson et al. PHYSIOLOGICAL REVIEWS
- Oral Delivery of Antisense Oligonucleotides in Man
- (2007) Lloyd G. Tillman et al. JOURNAL OF PHARMACEUTICAL SCIENCES
- Spinocerebellar ataxia type 7 (SCA7): widespread brain damage in an adult-onset patient with progressive visual impairments in comparison with an adult-onset patient without visual impairments
- (2007) U. Rüb et al. NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started