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Exosome-mediated delivery of siRNA in vitro and in vivo

Nature protocols, 2012-12, Vol.7 (12), p.2112-2126

2012

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Heterogeneity and interplay of the extracellular vesicle small RNA transcriptome and proteome

Scientific reports, 2018-07, Vol.8 (1), p.10813-12, Article 10813

2018

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Mutation-independent Proteomic Signatures of Pathological Progression in Murine Models of Duchenne Muscular Dystrophy

Molecular & cellular proteomics, 2020-12, Vol.19 (12), p.2047-2068

2020

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Alpha-synuclein release by neurons activates the inflammatory response in a microglial cell line

Neuroscience research, 2011-04, Vol.69 (4), p.337-342

2011

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Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

Molecular therapy. Nucleic acids, 2018-12, Vol.13, p.1-15

2018

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Advances in oligonucleotide drug delivery

Nature reviews. Drug discovery, 2020-10, Vol.19 (10), p.673-694

2020

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Cells release subpopulations of exosomes with distinct molecular and biological properties

Scientific reports, 2016-03, Vol.6 (1), p.22519-22519, Article 22519

2016

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Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Proceedings of the National Academy of Sciences - PNAS, 2016-09, Vol.113 (39), p.10962-10967

2016

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Alpha-synuclein induces the unfolded protein response in Parkinson's disease SNCA triplication iPSC-derived neurons

Human molecular genetics, 2017-11, Vol.26 (22), p.4441-4450

2017

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Tsc1 (hamartin) confers neuroprotection against ischemia by inducing autophagy

Nature medicine, 2013-03, Vol.19 (3), p.351-357

2013

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Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

Human molecular genetics, 2013-12, Vol.22 (24), p.4914-4928

2013

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Assessment of RT-qPCR normalization strategies for accurate quantification of extracellular microRNAs in murine serum

PloS one, 2014-02, Vol.9 (2), p.e89237-e89237

2014

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Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1

International journal of molecular sciences, 2023-01, Vol.24 (3), p.2697

2023

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Molecular and electrophysiological features of spinocerebellar ataxia type seven in induced pluripotent stem cells

PloS one, 2021-02, Vol.16 (2), p.e0247434-e0247434

2021

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Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

Gene therapy, 2019-11, Vol.27 (10-11), p.505-515

2019

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Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts

European journal of human genetics : EJHG, 2014-12, Vol.22 (12), p.1369-1375

2014

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Mirtrons, an emerging class of atypical miRNA

Wiley interdisciplinary reviews. RNA, 2012-09, Vol.3 (5), p.617-632

2012

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Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

Scientific reports, 2015-03, Vol.5 (1), p.8986-8986, Article 8986

2015

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RISC in PD: the impact of microRNAs in Parkinson's disease cellular and molecular pathogenesis

Frontiers in molecular neuroscience, 2013-11, Vol.6, p.40-40

2013

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

PloS one, 2009-09, Vol.4 (9), p.e7232

2009

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Detection and quantification of extracellular microRNAs in murine biofluids

Biological procedures online, 2014-03, Vol.16 (1), p.5-5, Article 5

2014

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Profiling of Extracellular Small RNAs Highlights a Strong Bias towards Non-Vesicular Secretion

Cells (Basel, Switzerland), 2021-06, Vol.10 (6), p.1543

2021

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Exosome-mediated delivery of siRNA in vitro and in vivo

Heterogeneity and interplay of the extracellular vesicle small RNA transcriptome and proteome

Mutation-independent Proteomic Signatures of Pathological Progression in Murine Models of Duchenne Muscular Dystrophy

Alpha-synuclein release by neurons activates the inflammatory response in a microglial cell line

Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

Advances in oligonucleotide drug delivery

Cells release subpopulations of exosomes with distinct molecular and biological properties

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Alpha-synuclein induces the unfolded protein response in Parkinson's disease SNCA triplication iPSC-derived neurons

Tsc1 (hamartin) confers neuroprotection against ischemia by inducing autophagy

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

Assessment of RT-qPCR normalization strategies for accurate quantification of extracellular microRNAs in murine serum

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1

Molecular and electrophysiological features of spinocerebellar ataxia type seven in induced pluripotent stem cells

Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts

Mirtrons, an emerging class of atypical miRNA

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

RISC in PD: the impact of microRNAs in Parkinson's disease cellular and molecular pathogenesis

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

Detection and quantification of extracellular microRNAs in murine biofluids

Profiling of Extracellular Small RNAs Highlights a Strong Bias towards Non-Vesicular Secretion

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