(Q34303837)

1 August 2017

title

RNAi-based therapies for Huntington's disease: delivery challenges and opportunities (English)

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1 August 2017

RNAi-based therapies for Huntington’s disease: delivery challenges and opportunities (English)

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Neelima Mantha

1

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1 August 2017

Sudip K Das

series ordinal

2

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1 August 2017

Nandita G Das

series ordinal

3

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1 August 2017

publication date

1 September 2012

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1 August 2017

September 2012

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published in

Therapeutic delivery

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1 August 2017

volume

3

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1 August 2017

issue

9

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1 August 2017

page(s)

1061-1076

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

1 August 2017

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Transcriptional abnormalities in Huntington disease

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Sp1 and TAFII130 transcriptional activity disrupted in early Huntington's disease

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21 January 2018

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Multiple pathways contribute to the pathogenesis of Huntington disease

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Apoptosis in Huntington's disease.

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Pathophysiology of Huntington's disease: from huntingtin functions to potential treatments

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Potential drugs and methods for preventing or delaying the progression of Huntington's disease

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Pharmaceutical, cellular and genetic therapies for Huntington's disease

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Argonaute2 is the catalytic engine of mammalian RNAi

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Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells

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Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs

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siRNA vs. shRNA: similarities and differences

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Comparative assessment of siRNA and shRNA off target effects: what is slowing clinical development

21 January 2018

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Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7

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Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?

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Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway

21 January 2018

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A status report on RNAi therapeutics

21 January 2018

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RNAi therapeutics: principles, prospects and challenges

21 January 2018

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RNAi therapeutics for CNS disorders

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

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Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA.

21 January 2018

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rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse

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

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High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo

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

21 January 2018

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An evaluation of oligonucleotide-based therapeutic strategies for polyQ diseases

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1 reference

Chemical modification: the key to clinical application of RNA interference?

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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Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

21 January 2018

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siRNA therapeutics: big potential from small RNAs

21 January 2018

1 reference

siRNA and isRNA: two edges of one sword

21 January 2018

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The promises and pitfalls of RNA-interference-based therapeutics

21 January 2018

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Widespread changes in protein synthesis induced by microRNAs

21 January 2018

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3' UTR seed matches, but not overall identity, are associated with RNAi off-targets

21 January 2018

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A systematic analysis of the silencing effects of an active siRNA at all single-nucleotide mismatched target sites

21 January 2018

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Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease.

21 January 2018

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Expression profiling reveals off-target gene regulation by RNAi

21 January 2018

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Biological barriers to therapy with antisense and siRNA oligonucleotides

21 January 2018

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Knocking down barriers: advances in siRNA delivery

21 January 2018

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Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing

21 January 2018

1 reference

Selective gene silencing by viral delivery of short hairpin RNA

21 January 2018

1 reference

Reconstituted influenza virus envelopes as an efficient carrier system for cellular delivery of small-interfering RNAs.

21 January 2018

1 reference

Progress and problems with the use of viral vectors for gene therapy

21 January 2018

1 reference

Viral vectors: from virology to transgene expression

21 January 2018

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Baculovirus vector-mediated transfer of NIS gene into colon tumor cells for radionuclide therapy

21 January 2018

1 reference

Cationic lipids and polymers mediated vectors for delivery of siRNA.

21 January 2018

1 reference

Nonviral delivery of synthetic siRNAs in vivo

21 January 2018

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Research progress on siRNA delivery with nonviral carriers

21 January 2018

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Efficient and safe delivery of siRNA using anionic lipids: Formulation optimization studies

21 January 2018

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Strategies for targeted nonviral delivery of siRNAs in vivo

21 January 2018

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siRNA-containing liposomes modified with polyarginine effectively silence the targeted gene

21 January 2018

1 reference

Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle

21 January 2018

1 reference

Recent advances of siRNA delivery by nanoparticles

21 January 2018

1 reference

Cell-penetrating peptides: classes, origin, and current landscape

21 January 2018

1 reference

Cell-penetrating peptides for the delivery of nucleic acids

21 January 2018

1 reference

Comparison of cationic and amphipathic cell penetrating peptides for siRNA delivery and efficacy

21 January 2018

1 reference

Blood-brain barrier P450 enzymes and multidrug transporters in drug resistance: a synergistic role in neurological diseases.

21 January 2018

1 reference

Drug delivery across the blood-brain barrier: why is it difficult? how to measure and improve it?

21 January 2018

1 reference

Liposome-siRNA-peptide complexes cross the blood-brain barrier and significantly decrease PrP on neuronal cells and PrP in infected cell cultures

21 January 2018

1 reference

SiRNA delivery with exosome nanoparticles.

21 January 2018

1 reference

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

21 January 2018

1 reference

Delivery of small-interfering RNA (siRNA) to the brain

21 January 2018

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Intranasal delivery to the central nervous system: mechanisms and experimental considerations

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.4155/TDE.12.80

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