A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.
about
Emerging role of non-coding RNA in neural plasticity, cognitive function, and neuropsychiatric disordersOligonucleotide therapeutic approaches for Huntington diseaseAllele-specific silencing of mutant huntingtin in rodent brain and human stem cellsSilencing mutant ataxin-3 rescues motor deficits and neuropathology in Machado-Joseph disease transgenic miceEpigenetic mechanisms of neurodegeneration in Huntington's diseaseAllele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patientsTargeting several CAG expansion diseases by a single antisense oligonucleotideThe expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain.Huntington's disease: silencing a brutal killer.Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing.Oligonucleotide-based strategies to combat polyglutamine diseases.Efficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi.An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseasesPotent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin.Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat.Tailor-made RNAi knockdown against triplet repeat disease-causing alleles.Targets for future clinical trials in Huntington's disease: what's in the pipeline?Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.Mesenchymal stem cells for the treatment of neurodegenerative disease.Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts.Antisense therapy in neurologyRNAi medicine for the brain: progresses and challenges.Aberrant RNA homeostasis in amyotrophic lateral sclerosis: potential for new therapeutic targets?Gene therapy in mouse models of huntington disease.Allele-specific knockdown of ALS-associated mutant TDP-43 in neural stem cells derived from induced pluripotent stem cellsSplice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6.Genetically engineered mesenchymal stem cells as a proposed therapeutic for Huntington's diseaseSix-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum.Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.Allele-selective inhibition of trinucleotide repeat genes.Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathwayAllele dependent silencing of collagen type I using small interfering RNAs targeting 3'UTR Indels - a novel therapeutic approach in osteogenesis imperfecta.Huntington disease in the South African population occurs on diverse and ethnically distinct genetic haplotypesExamination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for reduction of huntingtin.Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.Mass spectrometric identification of novel posttranslational modification sites in Huntingtin.Huntington's disease: progress toward effective disease-modifying treatments and a cureMolecular mechanisms and potential therapeutical targets in Huntington's disease.
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P2860
A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh-hant
name
A majority of Huntington's dis ...... ele-specific RNA interference.
@en
A majority of Huntington's dis ...... ele-specific RNA interference.
@nl
type
label
A majority of Huntington's dis ...... ele-specific RNA interference.
@en
A majority of Huntington's dis ...... ele-specific RNA interference.
@nl
prefLabel
A majority of Huntington's dis ...... ele-specific RNA interference.
@en
A majority of Huntington's dis ...... ele-specific RNA interference.
@nl
P2093
P50
P1476
A majority of Huntington's dis ...... lele-specific RNA interference
@en
P2093
Christine Spronkmans
Leonie Jaspers
Maria Stella Lombardi
Stefano Di Donato
William F Kaemmerer
P304
P356
10.1016/J.EXPNEUROL.2009.03.004
P407
P577
2009-03-13T00:00:00Z