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Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription

Scientific reports, 2022-06, Vol.12 (1), p.9674-9674, Article 9674

2022

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RFX1 and RFX3 Transcription Factors Interact with the D Sequence of Adeno-Associated Virus Inverted Terminal Repeat and Regulate AAV Transduction

Scientific reports, 2018-01, Vol.8 (1), p.210-210, Article 210

2018

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Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles

Human molecular genetics, 2016-08, Vol.25 (16), p.3555-3563

2016

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Actin scaffolding by clathrin heavy chain is required for skeletal muscle sarcomere organization

The Journal of cell biology, 2014-05, Vol.205 (3), p.377-393

2014

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Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy

Human gene therapy, 2016-09, Vol.27 (9), p.712-726

2016

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Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.695-708

2020

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miR-708-5p and miR-34c-5p are involved in nNOS regulation in dystrophic context

Skeletal muscle, 2018-04, Vol.8 (1), p.15-15, Article 15

2018

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An embryonic CaVβ1 isoform promotes muscle mass maintenance via GDF5 signaling in adult mouse

Science translational medicine, 2019-11, Vol.11 (517)

2019

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Blockade of ActRIIB Signaling Triggers Muscle Fatigability and Metabolic Myopathy

Molecular therapy, 2014-08, Vol.22 (8), p.1423-1433

2014

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Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions

The Journal of biological chemistry, 2018-05, Vol.293 (18), p.6637-6646

2018

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Is adipose tissue a place for Mycobacterium tuberculosis persistence?

PloS one, 2006-12, Vol.1 (1), p.e43-e43

2006

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New Insights in CaVβ Subunits: Role in the Regulation of Gene Expression and Cellular Homeostasis

Frontiers in cell and developmental biology, 2022-04, Vol.10, p.880441-880441

2022

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Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical rat model identifies COMP as biomarker of fibrosis

Acta neuropathologica communications, 2022-04, Vol.10 (1), p.60-60, Article 60

2022

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Variable phenotype of del45-55 Becker patients correlated with nNOSμ mislocalization and RYR1 hypernitrosylation

Human molecular genetics, 2012-08, Vol.21 (15), p.3449-3460

2012

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RhoA Is a Crucial Regulator of Myoblast Fusion

Cells (Basel, Switzerland), 2023-11, Vol.12 (23), p.2673

2023

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

EMBO molecular medicine, 2017-05, Vol.9 (5), p.545-557

2017

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Human adipose cells express CD4, CXCR4, and CCR5 receptors: a new target cell type for the immunodeficiency virus‐1?

The FASEB journal, 2002-08, Vol.16 (10), p.1254-1256

2002

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Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy

Human gene therapy, 2013-07, Vol.24 (7), p.702-713

2013

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CD38‐NADase is a new major contributor to Duchenne muscular dystrophic phenotype

EMBO molecular medicine, 2022-05, Vol.14 (5), p.e12860-n/a

2022

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The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMD

Biomedicines, 2021-02, Vol.9 (2), p.219

2021

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Functional Characterization and Rescue of a Deep Intronic Mutation in OCRL Gene Responsible for Lowe Syndrome

Human mutation, 2017-02, Vol.38 (2), p.152-159

2017

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Is oxygen a key factor in the lipodystrophy phenotype?

Lipids in health and disease, 2006-10, Vol.5 (1), p.27-27, Article 27

2006

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DHPR α1S subunit controls skeletal muscle mass and morphogenesis

The EMBO journal, 2010-02, Vol.29 (3), p.643-654

2010

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Progressive cardiomyopathy with intercalated disc disorganization in a rat model of Becker dystrophy

EMBO reports, 2024-10

2024

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Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription

RFX1 and RFX3 Transcription Factors Interact with the D Sequence of Adeno-Associated Virus Inverted Terminal Repeat and Regulate AAV Transduction

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles

Actin scaffolding by clathrin heavy chain is required for skeletal muscle sarcomere organization

Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy

Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice

miR-708-5p and miR-34c-5p are involved in nNOS regulation in dystrophic context

An embryonic CaVβ1 isoform promotes muscle mass maintenance via GDF5 signaling in adult mouse

Blockade of ActRIIB Signaling Triggers Muscle Fatigability and Metabolic Myopathy

Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions

Is adipose tissue a place for Mycobacterium tuberculosis persistence?

New Insights in CaVβ Subunits: Role in the Regulation of Gene Expression and Cellular Homeostasis

Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical rat model identifies COMP as biomarker of fibrosis

Variable phenotype of del45-55 Becker patients correlated with nNOSμ mislocalization and RYR1 hypernitrosylation

RhoA Is a Crucial Regulator of Myoblast Fusion

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

Human adipose cells express CD4, CXCR4, and CCR5 receptors: a new target cell type for the immunodeficiency virus‐1?

Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy

CD38‐NADase is a new major contributor to Duchenne muscular dystrophic phenotype

The lncRNA 44s2 Study Applicability to the Design of 45-55 Exon Skipping Therapeutic Strategy for DMD

Functional Characterization and Rescue of a Deep Intronic Mutation in OCRL Gene Responsible for Lowe Syndrome

Is oxygen a key factor in the lipodystrophy phenotype?

NR1D1 controls skeletal muscle calcium homeostasis through myoregulin repression

DHPR α1S subunit controls skeletal muscle mass and morphogenesis

Progressive cardiomyopathy with intercalated disc disorganization in a rat model of Becker dystrophy

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