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Genetics and Emerging Treatments for Duchenne and Becker Muscular Dystrophy

The Pediatric clinics of North America, 2015-06, Vol.62 (3), p.723-742

2015

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Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatment

Frontiers in cellular neuroscience, 2023-10, Vol.17, p.1248922-1248922

2023

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Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

Molecular therapy. Nucleic acids, 2021-03, Vol.23, p.476-486

2021

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Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

Molecular therapy. Methods & clinical development, 2021-06, Vol.21, p.325-340

2021

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Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45

Annals of neurology, 2015-04, Vol.77 (4), p.668-674

2015

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Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping

Human mutation, 2010-02, Vol.31 (2), p.136-142

2010

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Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders

Journal of personalized medicine, 2022-11, Vol.12 (12), p.1979

2022

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A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy

Science translational medicine, 2010-09, Vol.2 (50), p.50ra69

2010

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Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

Molecular therapy. Methods & clinical development, 2022-09, Vol.26, p.279-293

2022

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Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

Molecular therapy. Nucleic acids, 2022-12, Vol.30, p.479-492

2022

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Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

Molecular therapy. Methods & clinical development, 2023-12, Vol.31, p.101144-101144, Article 101144

2023

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Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines

Frontiers in cell and developmental biology, 2023-06, Vol.11, p.1181040

2023

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X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation

Skeletal muscle, 2020-08, Vol.10 (1), p.23-23, Article 23

2020

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The ZZ Domain of Dystrophin in DMD: Making Sense of Missense Mutations

Human mutation, 2014-02, Vol.35 (2), p.257-264

2014

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Personalized gene and cell therapy for Duchenne Muscular Dystrophy

Neuromuscular disorders : NMD, 2018-10, Vol.28 (10), p.803-824

2018

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U7 snRNA, a Small RNA with a Big Impact in Gene Therapy

Human gene therapy, 2021-11, Vol.32 (21-22), p.1317

2021

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Performance Requirements of Membrane Reactors for the Application in Renewable Methanol Synthesis: A Techno‐Economic Assessment

Advanced Sustainable Systems, 2022-12, Vol.6 (12), p.n/a

2022

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Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications

Human gene therapy, 2021-11, Vol.32 (21-22), p.1346

2021

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Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

Human gene therapy, 2021-09, Vol.32 (17-18), p.882

2021

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Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates

Molecular therapy, 2023-09, Vol.31 (9), p.2767-2782

2023

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Translational research and therapeutic perspectives in dysferlinopathies

Molecular medicine (Cambridge, Mass.), 2011-09, Vol.17 (9-10), p.875-882

2011

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Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy

Cell reports (Cambridge), 2022-12, Vol.41 (10), p.111751-111751, Article 111751

2022

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Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

Molecular therapy. Nucleic acids, 2021-03, Vol.23, p.476-486

2021

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Genetics and Emerging Treatments for Duchenne and Becker Muscular Dystrophy

Neuron-Schwann cell interactions in peripheral nervous system homeostasis, disease, and preclinical treatment

Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45

Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping

Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders

A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines

X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation

The ZZ Domain of Dystrophin in DMD: Making Sense of Missense Mutations

Personalized gene and cell therapy for Duchenne Muscular Dystrophy

U7 snRNA, a Small RNA with a Big Impact in Gene Therapy

Performance Requirements of Membrane Reactors for the Application in Renewable Methanol Synthesis: A Techno‐Economic Assessment

Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping

Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

Translational research and therapeutic perspectives in dysferlinopathies

Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy

Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

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