标题
Personalized gene silencing therapeutics for Huntington disease
作者
关键词
-
出版物
CLINICAL GENETICS
Volume 86, Issue 1, Pages 29-36
出版商
Wiley
发表日期
2014-03-20
DOI
10.1111/cge.12385
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。- High frequency of intermediate alleles on huntington disease-associated haplotypes in British Columbia's general population
- (2013) Alicia Semaka et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART B-NEUROPSYCHIATRIC GENETICS
- Huntington disease in the South African population occurs on diverse and ethnically distinct genetic haplotypes
- (2013) Fiona K Baine et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression
- (2013) Agnieszka Fiszer et al. NUCLEIC ACIDS RESEARCH
- 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
- Common SNP-Based Haplotype Analysis of the 4p16.3 Huntington Disease Gene Region
- (2012) Jong-Min Lee et al. AMERICAN JOURNAL OF HUMAN GENETICS
- 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
- A fully humanized transgenic mouse model of Huntington disease
- (2012) Amber L. Southwell et al. HUMAN MOLECULAR GENETICS
- Network Organization of the Huntingtin Proteomic Interactome in Mammalian Brain
- (2012) Dyna I. Shirasaki et al. NEURON
- Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
- (2012) Holly B. Kordasiewicz et al. NEURON
- Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases
- (2012) Amber L. Southwell et al. TRENDS IN MOLECULAR MEDICINE
- A Pathogenic Mechanism in Huntington's Disease Involves Small CAG-Repeated RNAs with Neurotoxic Activity
- (2012) Mónica Bañez-Coronel et al. PLoS Genetics
- HTT haplotypes contribute to differences in Huntington disease prevalence between Europe and East Asia
- (2011) Simon C Warby et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- Silencing disease genes in the laboratory and the clinic
- (2011) Jonathan K Watts et al. JOURNAL OF PATHOLOGY
- 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
- Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
- (2011) Nathalie M. Goemans et al. NEW ENGLAND JOURNAL OF MEDICINE
- Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats
- (2011) Agnieszka Fiszer et al. NUCLEIC ACIDS RESEARCH
- 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
- Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver
- (2010) Dirk Grimm et al. JOURNAL OF CLINICAL INVESTIGATION
- Novel RNA-based Strategies for Therapeutic Gene Silencing
- (2010) Christopher R Sibley et al. MOLECULAR THERAPY
- Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease
- (2010) Chiara Zuccato et al. PHYSIOLOGICAL REVIEWS
- 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
- A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference
- (2009) Maria Stella Lombardi et al. EXPERIMENTAL NEUROLOGY
- Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs
- (2009) Jiaxin Hu et al. NATURE BIOTECHNOLOGY
- Triplet repeat length bias and variation in the human transcriptome
- (2009) M. Molla et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- 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
- Phylogenetic Comparison of Huntingtin Homologues Reveals the Appearance of a Primitive polyQ in Sea Urchin
- (2007) M. Tartari et al. MOLECULAR BIOLOGY AND EVOLUTION
Discover Peeref hubs
Discuss science. Find collaborators. Network.
Join a conversationAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now