Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease
about
Emerging role of non-coding RNA in neural plasticity, cognitive function, and neuropsychiatric disordersMind the gap: models in multiple species needed for therapeutic development in Huntington's diseaseMechanisms of RNA-induced toxicity in CAG repeat disordersPluripotent stem cells models for Huntington's disease: prospects and challengesAAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patientsA fully humanized transgenic mouse model of Huntington disease.Quantification assays for total and polyglutamine-expanded huntingtin proteins.Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing.Use of Genetically Altered Stem Cells for the Treatment of Huntington's Disease.Oligonucleotide-based strategies to combat polyglutamine diseases.Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.mRNA-Seq and microRNA-Seq whole-transcriptome analyses of rhesus monkey embryonic stem cell neural differentiation revealed the potential regulators of rosette neural stem cells.Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapysiSPOTR: a tool for designing highly specific and potent siRNAs for human and mouse.Targets for future clinical trials in Huntington's disease: what's in the pipeline?Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.Reversal of cellular phenotypes in neural cells derived from Huntington's disease monkey-induced pluripotent stem cellsBroad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy.Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain.Selection of appropriate reference genes for RT-qPCR analysis in a streptozotocin-induced Alzheimer's disease model of cynomolgus monkeys (Macaca fascicularis).The role of the immune system in triplet repeat expansion diseasesGene therapy for the nervous system: challenges and new strategies.miR-888: A Novel Cancer-Testis Antigen that Targets the Progesterone Receptor in Endometrial Cancer.Progressive cognitive deficit, motor impairment and striatal pathology in a transgenic Huntington disease monkey model from infancy to adulthood.Thiol-disulfide Oxidoreductases TRX1 and TMX3 Decrease Neuronal Atrophy in a Lentiviral Mouse Model of Huntington's DiseaseTherapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy.Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA.Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice.Epigenetics and the transition from acute to chronic pain.Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infectionPotential trade-offs in treatment of premanifest Huntington's disease.Recent advances in RNA interference therapeutics for CNS diseases.Gene therapy for misfolding protein diseases of the central nervous system.Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease.Toward RNAi therapy for the polyglutamine disease Machado-Joseph diseaseSafety of Striatal Infusion of siRNA in a Transgenic Huntington's Disease Mouse Model.Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.Replacement of huntingtin exon 1 by trans-splicing.
P2860
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P2860
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease
description
2011 nî lūn-bûn
@nan
2011 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@ast
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@en
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@nl
type
label
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@ast
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@en
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@nl
prefLabel
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@ast
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@en
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@nl
P2093
P2860
P3181
P356
P1433
P1476
Preclinical safety of RNAi-med ...... erapy for Huntington's disease
@en
P2093
Beverly L Davidson
Brett Dufour
Jodi L McBride
Mark R Pitzer
Ryan L Boudreau
Sergio R Ojeda
Theodore Hobbs
P2860
P304
P3181
P356
10.1038/MT.2011.219
P407
P577
2011-12-01T00:00:00Z