Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing (Q38244104)

31 August 2017

title

Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing (English)

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31 August 2017

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Neil Aronin

1

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31 August 2017

Marian DiFiglia

series ordinal

2

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31 August 2017

language of work or name

English

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publication date

27 August 2014

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31 August 2017

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31 August 2017

volume

29

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31 August 2017

issue

11

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31 August 2017

page(s)

1455-1461

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An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

31 August 2017

cites work

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

21 January 2018

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Oligonucleotide therapeutic approaches for Huntington disease

21 January 2018

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Dominant effects of the Huntington's disease HTT CAG repeat length are captured in gene-expression data sets by a continuous analysis mathematical modeling strategy

21 January 2018

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De novo Huntington disease caused by 26-44 CAG repeat expansion on a low-risk haplotype

21 January 2018

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Relationship between trinucleotide repeat expansion and phenotypic variation in Huntington's disease

21 January 2018

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CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion.

21 January 2018

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21 January 2018

MicroRNA therapeutics.

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Small silencing RNAs: an expanding universe

21 January 2018

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Kinetic analysis of the RNAi enzyme complex

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RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

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Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

21 January 2018

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Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin

21 January 2018

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Asymmetry in the assembly of the RNAi enzyme complex

21 January 2018

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Expression profiling reveals off-target gene regulation by RNAi

21 January 2018

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Target selectivity in mRNA silencing

21 January 2018

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Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.

21 January 2018

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MicroRNAs: target recognition and regulatory functions

21 January 2018

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Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits

21 January 2018

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Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients

21 January 2018

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A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.

21 January 2018

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Regulation of postsynaptic retrograde signaling by presynaptic exosome release

21 January 2018

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Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes

21 January 2018

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Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.

21 January 2018

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Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi

21 January 2018

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Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

21 January 2018

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Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system

21 January 2018

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Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

21 January 2018

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Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach

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rAAV human trial experience

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Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

21 January 2018

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RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

21 January 2018

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Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice

21 January 2018

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

21 January 2018

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Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response

21 January 2018

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Spt4 is selectively required for transcription of extended trinucleotide repeats

21 January 2018

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Paradoxical delay in the onset of disease caused by super-long CAG repeat expansions in R6/2 mice.

21 January 2018

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CAG repeat lengths > or =335 attenuate the phenotype in the R6/2 Huntington's disease transgenic mouse

21 January 2018

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A critical window of CAG repeat-length correlates with phenotype severity in the R6/2 mouse model of Huntington's disease

21 January 2018

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A guide to genome engineering with programmable nucleases

21 January 2018

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TALENs: a widely applicable technology for targeted genome editing

21 January 2018

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Sequence- and Structure-Specific RNA Processing by a CRISPR Endonuclease

21 January 2018

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Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice

21 January 2018

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Cas9 as a versatile tool for engineering biology

21 January 2018

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Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression

21 January 2018

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DNA interrogation by the CRISPR RNA-guided endonuclease Cas9.

21 January 2018

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Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue

21 January 2018

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Wild-type huntingtin plays a role in brain development and neuronal survival

21 January 2018

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HTT-lowering reverses Huntington's disease immune dysfunction caused by NFκB pathway dysregulation

21 January 2018

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21 January 2018

Identifiers

DOI

10.1002/MDS.26020

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31 August 2017

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