Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease - Test2 - elhamkhani - TriplyDB

Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease

Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease

http://www.wikidata.org/entity/Q24626259

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Emerging role of non-coding RNA in neural plasticity, cognitive function, and neuropsychiatric disorders Mind the gap: models in multiple species needed for therapeutic development in Huntington's disease Mechanisms of RNA-induced toxicity in CAG repeat disorders Pluripotent stem cells models for Huntington's disease: prospects and challenges AAV 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 patients A 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 therapy siSPOTR: 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 cells Broad 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 diseases Gene 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 Disease Therapeutic 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 infection Potential 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 disease Safety 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.

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Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease

http://www.wikidata.org/entity/Q24626259

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2011 nî lūn-bûn

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2011年の論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年论文

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Molecular Therapy

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Preclinical safety of RNAi-med ...... erapy for Huntington's disease

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Beverly L Davidson

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Brett Dufour

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Jodi L McBride

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Mark R Pitzer

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Ryan L Boudreau

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Sergio R Ojeda

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Theodore Hobbs

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P2860

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes.

MicroRNAs: small RNAs with a big role in gene regulation

Huntingtin interacts with REST/NRSF to modulate the transcription of NRSE-controlled neuronal genes

Inactivation of the mouse Huntington's disease gene homolog Hdh

AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease

Cholinergic neuronal defect without cell loss in Huntington's disease.

A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells

Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.

Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates

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2152-62

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403877

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10.1038/MT.2011.219

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12

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19

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2011-12-01T00:00:00Z

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51749024

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22031240

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Huntington disease

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3242667

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