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Cas9 targeting of toxic foci of RNA repeats

Nature biomedical engineering, 2021-02, Vol.5 (2), p.130-131

Furling, Denis

2021

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Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds

Disease models & mechanisms, 2017-04, Vol.10 (4), p.487-497

2017

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Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice

Molecular therapy, 2019-08, Vol.27 (8), p.1372-1388

2019

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Splicing misregulation of SCN5A contributes to cardiac-conduction delay and heart arrhythmia in myotonic dystrophy

Nature communications, 2016-04, Vol.7 (1), p.11067-11067, Article 11067

2016

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Steroid receptor RNA activator protein binds to and counteracts SRA RNA-mediated activation of MyoD and muscle differentiation

Nucleic acids research, 2011-01, Vol.39 (2), p.513-525

2011

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Alternative splicing of clathrin heavy chain contributes to the switch from coated pits to plaques

The Journal of cell biology, 2020-09, Vol.219 (9), p.1

2020

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Targeting deregulated AMPK/mTORC1 pathways improves muscle function in myotonic dystrophy type I

The Journal of clinical investigation, 2017-02, Vol.127 (2), p.549-563

2017

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A CRISPR-Cas13a Based Strategy That Tracks and Degrades Toxic RNA in Myotonic Dystrophy Type 1

Frontiers in genetics, 2020-12, Vol.11, p.594576

2020

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The beneficial effect of chronic muscular exercise on muscle fragility is increased by Prox1 gene transfer in dystrophic mdx muscle

PloS one, 2022-04, Vol.17 (4), p.e0254274-e0254274

2022

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CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing

Molecular therapy, 2017-01, Vol.25 (1), p.24-43

2017

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Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

Nucleic acids research, 2018-09, Vol.46 (16), p.8275-8298

2018

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Improvement of Dystrophic Muscle Fragility by Short-Term Voluntary Exercise through Activation of Calcineurin Pathway in mdx Mice

The American journal of pathology, 2018-11, Vol.188 (11), p.2662-2673

2018

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rbFOX1/MBNL1 competition for CCUG RNA repeats binding contributes to myotonic dystrophy type 1/type 2 differences

Nature communications, 2018-05, Vol.9 (1), p.2009-15, Article 2009

2018

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Expression of miR-1, miR-133a, miR-133b and miR-206 increases during development of human skeletal muscle

BMC developmental biology, 2011-06, Vol.11 (1), p.34-34, Article 34

2011

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Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

Proceedings of the National Academy of Sciences - PNAS, 2009-08, Vol.106 (33), p.13915-13920

2009

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Dysregulation of Circular RNAs in Myotonic Dystrophy Type 1

International journal of molecular sciences, 2019-04, Vol.20 (8), p.1938

2019

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Cardiovascular manifestations of myotonic dystrophy

Trends in cardiovascular medicine, 2020-05, Vol.30 (4), p.232-238

2020

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Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus

PLoS genetics, 2012-11, Vol.8 (11), p.e1003043

2012

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Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs

Nature structural & molecular biology, 2011-01, Vol.18 (1), p.85-87

2011

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RBFOX1 cooperates with MBNL1 to control splicing in muscle, including events altered in myotonic dystrophy type 1

PloS one, 2014-09, Vol.9 (9), p.e107324-e107324

2014

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In-depth analysis of the secretome identifies three major independent secretory pathways in differentiating human myoblasts

Journal of proteomics, 2012-12, Vol.77, p.344-356

2012

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Misregulation of alternative splicing and microRNA processing in DM1 pathogenesis

Rinsho shinkeigaku = Clinical neurology, 2012, Vol.52 (11), p.1018-1022

Furling, Denis

2012

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Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells

Molecular therapy. Nucleic acids, 2023-06, Vol.32, p.857-871

2023

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Human circulating AC133+ stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle

The Journal of clinical investigation, 2004-07, Vol.114 (2), p.182-195

2004

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Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle

The Journal of clinical investigation, 2004-07, Vol.114 (2), p.182-195

2004

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Cas9 targeting of toxic foci of RNA repeats

Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds

Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice

Splicing misregulation of SCN5A contributes to cardiac-conduction delay and heart arrhythmia in myotonic dystrophy

Steroid receptor RNA activator protein binds to and counteracts SRA RNA-mediated activation of MyoD and muscle differentiation

Alternative splicing of clathrin heavy chain contributes to the switch from coated pits to plaques

Targeting deregulated AMPK/mTORC1 pathways improves muscle function in myotonic dystrophy type I

A CRISPR-Cas13a Based Strategy That Tracks and Degrades Toxic RNA in Myotonic Dystrophy Type 1

The beneficial effect of chronic muscular exercise on muscle fragility is increased by Prox1 gene transfer in dystrophic mdx muscle

CRISPR/Cas9-Induced (CTG⋅CAG)n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing

Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

Improvement of Dystrophic Muscle Fragility by Short-Term Voluntary Exercise through Activation of Calcineurin Pathway in mdx Mice

rbFOX1/MBNL1 competition for CCUG RNA repeats binding contributes to myotonic dystrophy type 1/type 2 differences

Expression of miR-1, miR-133a, miR-133b and miR-206 increases during development of human skeletal muscle

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

Dysregulation of Circular RNAs in Myotonic Dystrophy Type 1

Cardiovascular manifestations of myotonic dystrophy

Molecular, physiological, and motor performance defects in DMSXL mice carrying >1,000 CTG repeats from the human DM1 locus

Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs

RBFOX1 cooperates with MBNL1 to control splicing in muscle, including events altered in myotonic dystrophy type 1

In-depth analysis of the secretome identifies three major independent secretory pathways in differentiating human myoblasts

Misregulation of alternative splicing and microRNA processing in DM1 pathogenesis

Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells

Human circulating AC133+ stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle

Human circulating AC133(+) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle

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