Delivery is key: lessons learnt from developing splice-switching antisense therapies - Wikidata - awgldk - TriplyDB

Delivery is key: lessons learnt from developing splice-switching antisense therapies

Delivery is key: lessons learnt from developing splice-switching antisense therapies

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Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Natural antisense transcripts in diseases: From modes of action to targeted therapies.Impact, Characterization, and Rescue of Pre-mRNA Splicing Mutations in Lysosomal Storage Disorders.Oligonucleotide therapeutics in neurodegenerative diseases.The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders.Assisted delivery of antisense therapeutics in animal models of heritable neurodegenerative and neuromuscular disorders: a systematic review and meta-analysis.Antisense Phosphorodiamidate Morpholino Oligomers as Novel Antiviral Compounds.Recent Advances in Antisense Oligonucleotide Therapy in Genetic Neuromuscular Diseases.Structure-activity relationships and cellular mechanism of action of small molecules that enhance the delivery of oligonucleotides.A flow platform for degradation-free CuAAC bioconjugation Cationic Oligospermine-Oligonucleotide Conjugates Provide Carrier-free Splice Switching in Monolayer Cells and Spheroids

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P2860

Delivery is key: lessons learnt from developing splice-switching antisense therapies

http://www.wikidata.org/entity/Q33625875

description

2017 nî lūn-bûn

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2017 թուականի Մայիսին հրատարակուած գիտական յօդուած

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2017 թվականի մայիսին հրատարակված գիտական հոդված

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2017年の論文

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2017年論文

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2017年論文

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2017年論文

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2017年論文

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2017年論文

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2017年论文

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name

Delivery is key: lessons learnt from developing splice-switching antisense therapies

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Delivery is key: lessons learnt from developing splice-switching antisense therapies

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type

label

Delivery is key: lessons learnt from developing splice-switching antisense therapies

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Delivery is key: lessons learnt from developing splice-switching antisense therapies

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prefLabel

Delivery is key: lessons learnt from developing splice-switching antisense therapies

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Delivery is key: lessons learnt from developing splice-switching antisense therapies

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EMBO Molecular Medicine

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Delivery is key: lessons learnt from developing splice-switching antisense therapies

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Bernard Khoo

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Bård Smedsrød

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Camilla Brolin

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Caroline Godfrey

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Lourdes R Desviat

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Rayan Anwar

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Samir El Andaloussi

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Valentina Sardone

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Willeke Mc van Roon-Mom

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Creative Commons Attribution

P2860

Peptides for nucleic acid delivery

Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.

Randomized dose-comparison studies of intravitreous fomivirsen for treatment of cytomegalovirus retinitis that has reactivated or is persistently active despite other therapies in patients with AIDS

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

Integrated Safety Assessment of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers

Sex and ApoE genotype differences in treatment response to two doses of intranasal insulin in adults with mild cognitive impairment or Alzheimer's disease.

Antisense oligonucleotide therapies: the promise and the challenges from a toxicologic pathologist's perspective.

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents.

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545-557

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scholarly article

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10.15252/EMMM.201607199

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5

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9

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Virginia Arechavala-Gomeza

Michela A. Denti

Stéphanie Lorain

Annemieke Aartsma-Rus

France Piétri-Rouxel

Aurélie Goyenvalle

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2017-03-13T00:00:00Z

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28289078

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regulation of gene expression

biomedical investigative technique

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5412803

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