(Q37871417)

24 August 2017

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title

RNAi: a potential new class of therapeutic for human genetic disease (English)

1 reference

24 August 2017

Attila A Seyhan

1

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24 August 2017

publication date

3 May 2011

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24 August 2017

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24 August 2017

volume

130

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24 August 2017

issue

5

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24 August 2017

page(s)

583-605

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https://scigraph.springernature.com/pub.10.1007/s00439-011-0995-8

24 August 2017

exact match

0 references

cites work

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Allele-specific silencing of a pathogenic mutant acetylcholine receptor subunit by RNA interference

21 January 2018

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Mice lacking alpha-synuclein display functional deficits in the nigrostriatal dopamine system

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Role of Toll-like receptors in antisense and siRNA [corrected].

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MicroRNAs as biomarkers in rheumatic diseases.

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MicroRNAs in Sjögren's syndrome as a prototypic autoimmune disease.

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Tolerance for mutations and chemical modifications in a siRNA.

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The functions of animal microRNAs

21 January 2018

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siRNA as a route to new cancer therapies

21 January 2018

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Development of gene-specific double-stranded RNA drugs

21 January 2018

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MicroRNAs: genomics, biogenesis, mechanism, and function

21 January 2018

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MicroRNAs: target recognition and regulatory functions

21 January 2018

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Delivery of therapeutic agents to the central nervous system: the problems and the possibilities.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Chemical modification of siRNAs for in vivo use.

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

DMPK dosage alterations result in atrioventricular conduction abnormalities in a mouse myotonic dystrophy model

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Fusion antibody for Alzheimer's disease with bidirectional transport across the blood-brain barrier and abeta fibril disaggregation

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Inhibitory RNA in epilepsy: research tools and therapeutic perspectives

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Stable suppression of tumorigenicity by virus-mediated RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi therapeutics: a potential new class of pharmaceutical drugs

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

MicroRNA-133 controls cardiac hypertrophy

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

The promises and pitfalls of RNA-interference-based therapeutics

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

YAC transgenic mice carrying pathological alleles of the MJD1 locus exhibit a mild and slowly progressive cerebellar deficit.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Transactivation of miR-34a by p53 broadly influences gene expression and promotes apoptosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Specialization and evolution of endogenous small RNA pathways

21 January 2018

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https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

microRNA modulation of circadian-clock period and entrainment

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Myotonic dystrophy: emerging mechanisms for DM1 and DM2.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Aptamer mediated siRNA delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Recessive inheritance and variable penetrance of slow-channel congenital myasthenic syndromes

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Induction of stable RNA interference in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Targeting oncogenic fusion genes in leukemias and lymphomas by RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA interference: a tool for querying nervous system function and an emerging therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Viral vectors for gene delivery to the nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Molecular medicine for the brain: silencing of disease genes with RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Interfering with disease: a progress report on siRNA-based therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Harnessing RNA interference to develop neonatal therapies: from Nobel Prize winning discovery to proof of concept clinical trials.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Rnai as an experimental and therapeutic tool to study and regulate physiological and disease processes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

siRNAs can function as miRNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Central delivery of Dicer-substrate siRNA: a direct application for pain research

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Sustained effects of nonallele-specific Huntingtin silencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Killing the messenger: short RNAs that silence gene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Determinants of specific RNA interference-mediated silencing of human beta-globin alleles differing by a single nucleotide polymorphism

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA interference is mediated by 21- and 22-nucleotide RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Molecular pathogenesis of Parkinson disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

MicroRNAs in development and disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Stage-specific sensitivity to p53 restoration during lung cancer progression.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA interference in cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Small silencing RNAs: an expanding universe

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi-mediated inhibition of cathepsin B and uPAR leads to decreased cell invasion, angiogenesis and tumor growth in gliomas.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Technology insight: therapeutic RNA interference--how far from the neurology clinic?

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB).

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

The Microprocessor complex mediates the genesis of microRNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Efficacy of gene therapy for X-linked severe combined immunodeficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

A species of small antisense RNA in posttranscriptional gene silencing in plants

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

AAV serotype 8-mediated gene delivery of a soluble VEGF receptor to the CNS for the treatment of glioblastoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

The amyloid hypothesis of Alzheimer's disease: progress and problems on the road to therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Postoperative complications in myotonic dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Progress and challenges in RNA interference therapy for Huntington disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

A microRNA polycistron as a potential human oncogene

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Positional effects of short interfering RNAs targeting the human coagulation trigger Tissue Factor

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Sequence-specific knockdown of EWS-FLI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing's sarcoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Initial sequencing and analysis of the human genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Expression profiling reveals off-target gene regulation by RNAi

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

AKT1 and AKT2 maintain hematopoietic stem cell function by regulating reactive oxygen species.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi as a new therapeutic strategy against HCV.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Functional siRNAs and miRNAs Exhibit Strand Bias

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi mechanisms and applications

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Argonaute-1 directs siRNA-mediated transcriptional gene silencing in human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Tumor therapy mediated by lentiviral expression of shBcl-2 and S-TRAIL.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Therapeutic application of small interfering RNA directed against bcr-abl transcripts to a patient with imatinib-resistant chronic myeloid leukaemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Modification of alternative splicing by antisense therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Sustained expression of Mect1-Maml2 is essential for tumor cell growth in salivary gland cancers carrying the t(11;19) translocation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Identification of novel genes coding for small expressed RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Identification of tissue-specific microRNAs from mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Therapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Cytoplasmic and nuclear retained DMPK mRNAs are targets for RNA interference in myotonic dystrophy cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Lymphoproliferative disorders: prospects for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Therapeutic siRNAs for dominant genetic skin disorders including pachyonychia congenita.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Abnormal Na channel gating in murine cardiac myocytes deficient in myotonic dystrophy protein kinase

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi in combination with a ribozyme and TAR decoy for treatment of HIV infection in hematopoietic cell gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

miR-181a is an intrinsic modulator of T cell sensitivity and selection

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Neurological abnormalities in a knock-in mouse model of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

siRNA-mediated off-target gene silencing triggered by a 7 nt complementation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Targeting neurological disease with RNAi.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

MicroRNA expression profiles classify human cancers

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Interactions between double-stranded RNA regulators and the protein kinase DAI

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Applications of RNA interference in mammalian systems.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Synthetic small inhibiting RNAs: efficient tools to inactivate oncogenic mutations and restore p53 pathways.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA interference-mediated silencing of mutant superoxide dismutase rescues cyclosporin A-induced death in cultured neuroblastoma cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Inhibition of hepatitis B virus in mice by RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Adeno-associated virus (AAV) vectors in the CNS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Gene silencing in mammals by small interfering RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Mechanisms of gene silencing by double-stranded RNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Allele-specific silencing of dominant disease genes

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Targeting Alzheimer's disease genes with RNA interference: an efficient strategy for silencing mutant alleles

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Skeletal muscle sodium channel gating in mice deficient in myotonic dystrophy protein kinase

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Neuropeptide Y overexpression using recombinant adeno-associated viral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

ATP requirements and small interfering RNA structure in the RNA interference pathway

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Enhancement of allele discrimination by introduction of nucleotide mismatches into siRNA in allele-specific gene silencing by RNAi

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Fluorescence correlation spectroscopy and fluorescence cross-correlation spectroscopy reveal the cytoplasmic origination of loaded nuclear RISC in vivo in human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Prospects of RNA interference therapy for cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Toward an understanding of polyglutamine neurodegeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Polyglutamine disease and neuronal cell death.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Correction of sickle cell disease in transgenic mouse models by gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

On the art of identifying effective and specific siRNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

In vivo RNAi: today and tomorrow

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Gene therapy strategies for hemophilia: benefits versus risks

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Lentivector-mediated RNAi efficiently suppresses prion protein and prolongs survival of scrapie-infected mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Mutation in the alpha-synuclein gene identified in families with Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA interference screening for the discovery of oncology targets

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Viral-based modelling and correction of neurodegenerative diseases by RNA interference.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

The 21-nucleotide let-7 RNA regulates developmental timing in Caenorhabditis elegans

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

21 January 2018

Gene therapy in epilepsy

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA helicase A interacts with RISC in human cells and functions in RISC loading

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Specific and potent RNAi in the nucleus of human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Identification of mammalian microRNA host genes and transcription units

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Allele-specific RNA interference for neurological disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Allele-specific RNAi mitigates phenotypic progression in a transgenic model of Alzheimer's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Therapeutic potential of RNA interference for neurological disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Silencing of human alpha-synuclein in vitro and in rat brain using lentiviral-mediated RNAi

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Small RNAs with imperfect match to endogenous mRNA repress translation. Implications for off-target activity of small inhibitory RNA in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Specific inhibition of bcr-abl gene expression by small interfering RNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Asymmetry in the assembly of the RNAi enzyme complex

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Designing siRNA that distinguish between genes that differ by a single nucleotide

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Gene therapy for BCR-ABL+ human CML with dual phosphorylation resistant p27Kip1 and stable RNA interference using an EBV vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi screening for the discovery of novel modulators of human disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Complete, gene-specific siRNA libraries: production and expression in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA interference-mediated inhibition of Semliki Forest virus replication in mammalian cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

alpha-Synuclein locus triplication causes Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNA interference and double-stranded-RNA-activated pathways

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Activation of the interferon system by short-interfering RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Alpha-synuclein in Lewy bodies

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Effective inhibition of cell growth and invasion of melanoma by combined suppression of BRAF (V599E) and Skp2 with lentiviral RNAi.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Suppression of hippocampal TRPM7 protein prevents delayed neuronal death in brain ischemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Therapeutic potential of RNA interference against cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Validation of met as a therapeutic target in alveolar and embryonal rhabdomyosarcoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Short double-stranded RNA induces transcriptional gene silencing in human cancer cells in the absence of DNA methylation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

A status report on RNAi therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

The Sequence of the Human Genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

shRNAs targeting hepatitis C: effects of sequence and structural features, and comparision with siRNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Importin 8 is a gene silencing factor that targets argonaute proteins to distinct mRNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Single treatment with RNAi against prion protein rescues early neuronal dysfunction and prolongs survival in mice with prion disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Killing of leukemic cells with a BCR/ABL fusion gene by RNA interference (RNAi).

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Inhibition of bcr-abl gene expression by small interfering RNA sensitizes for imatinib mesylate (STI571).

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Discovery, biology and therapeutic potential of RNA interference, microRNA and antagomirs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Ribo-gnome: the big world of small RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

MicroRNAs and their regulatory roles in animals and plants

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

High-throughput screening of effective siRNAs from RNAi libraries delivered via bacterial invasion

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Novel dual inhibitory function aptamer-siRNA delivery system for HIV-1 therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Enhancing TRAIL-induced apoptosis by Bcl-X(L) siRNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

RNAi-mediated gene silencing in non-human primates.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Glutamine repeats and neurodegeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

SCA1 transgenic mice: A model for neurodegeneration caused by an expanded CAG trinucleotide repeat

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

Cancer: The blind spot of p53

21 January 2018

1 reference

https://api.crossref.org/works/10.1007%2FS00439-011-0995-8

An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models

21 January 2018

1 reference

12 December 2020

RNA interference: a potential therapeutic tool for silencing splice isoforms linked to human diseases.

1 reference

12 December 2020

RNAi and gene therapy: a mutual attraction

1 reference

12 December 2020

Hydroxyurea-induced augmentation of fetal hemoglobin production in patients with sickle cell anemia.

1 reference

12 December 2020

Identification of essential genes in cultured mammalian cells using small interfering RNAs.

1 reference

12 December 2020

171st ENMC International Workshop: Standards of care and management of facioscapulohumeral muscular dystrophy

1 reference

12 December 2020

Peptide-oligonucleotide conjugates for the delivery of antisense and siRNA.

1 reference

12 December 2020

Harnessing RNA interference for the treatment of viral infections

1 reference

12 December 2020

Overview of gene silencing by RNA interference.

1 reference

12 December 2020

Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors

1 reference

12 December 2020

Genetic characterization of a large, historically significant Utah kindred with facioscapulohumeral dystrophy

1 reference

12 December 2020

The mechanism of naked DNA uptake and expression

1 reference

12 December 2020

MicroRNAs: biogenesis, function and applications.

1 reference

12 December 2020

Neurodegeneration with tau accumulation in a transgenic mouse expressing V337M human tau.

1 reference

12 December 2020

Mice lacking the myotonic dystrophy protein kinase develop a late onset progressive myopathy

1 reference

12 December 2020

Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA

1 reference

12 December 2020

Inhibition of Hepatitis C IRES-Mediated Gene Expression by Small Hairpin RNAs in Human Hepatocytes and Mice

1 reference

12 December 2020

Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model

1 reference

12 December 2020

RNA-mediated gene silencing in hematopoietic cells

1 reference

12 December 2020

Stable expression of small interfering RNA sensitizes TEL-PDGFbetaR to inhibition with imatinib or rapamycin.

1 reference

12 December 2020

Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation

1 reference

12 December 2020

Facioscapulohumeral muscular dystrophy

1 reference

12 December 2020

10.1007/S00439-011-0995-8

Identifiers

DOI

1 reference

24 August 2017

1 reference