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A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

Cell stem cell, 2016-04, Vol.18 (4), p.533-540

2016

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DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype

Human mutation, 2018-09, Vol.39 (9), p.1193-1202

2018

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Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype

The Journal of cell biology, 2016-04, Vol.213 (2), p.275-288

2016

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Quantitative immuno-mass spectrometry imaging of skeletal muscle dystrophin

Scientific reports, 2021-01, Vol.11 (1), p.1128-1128, Article 1128

2021

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PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy

Neurology, 2014-06, Vol.82 (23), p.2085-2091

2014

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De Novo Nonsense Mutations in KAT6A, a Lysine Acetyl-Transferase Gene, Cause a Syndrome Including Microcephaly and Global Developmental Delay

American journal of human genetics, 2015-03, Vol.96 (3), p.498-506

2015

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A well‐tolerated core needle muscle biopsy process suitable for children and adults

Muscle & nerve, 2020-12, Vol.62 (6), p.688-698

2020

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Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models

Molecular therapy. Nucleic acids, 2019-12, Vol.18, p.580-589

2019

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The case for eteplirsen: Paving the way for precision medicine

Molecular genetics and metabolism, 2016-06, Vol.118 (2), p.70-71

2016

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Discs Large Homolog 1 Splice Variants Regulate p38-Dependent and -Independent Effector Functions in CD8+ T Cells

PloS one, 2015-07, Vol.10 (7), p.e0133353

2015

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Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy

Science translational medicine, 2012-12, Vol.4 (164), p.164ra160

2012

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Transcriptomic analysis of paired healthy human skeletal muscles to identify modulators of disease severity in DMD

Frontiers in genetics, 2023-07, Vol.14, p.1216066-1216066

2023

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Single nuclei transcriptomics of muscle reveals intra-muscular cell dynamics linked to dystrophin loss and rescue

Communications biology, 2022-09, Vol.5 (1), p.989-989, Article 989

2022

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Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse

Molecular therapy. Nucleic acids, 2018-06, Vol.11 (C), p.180-191

2018

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Emerging genetic therapies to treat Duchenne muscular dystrophy

Current opinion in neurology, 2009-10, Vol.22 (5), p.532-538

2009

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Modeling Patient-Specific Muscular Dystrophy Phenotypes and Therapeutic Responses in Reprogrammed Myotubes Engineered on Micromolded Gelatin Hydrogels

Frontiers in cell and developmental biology, 2022-04, Vol.10, p.830415-830415

2022

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Characterization of in vivo Dlg1 deletion on T cell development and function

PloS one, 2012-09, Vol.7 (9), p.e45276-e45276

2012

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FDA Approval of Eteplirsen for Muscular Dystrophy

JAMA : the journal of the American Medical Association, 2017-04, Vol.317 (14), p.1480

2017

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Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta

The Journal of clinical investigation, 2009-06, Vol.119 (6), p.1583-1594

2009

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Validation and Detection of Exon Skipping Boosters in DMD Patient Cell Models and mdx Mouse

Methods in molecular biology (Clifton, N.J.), 2018, Vol.1828, p.309

2018

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Lectin-binding characterizes the healthy human skeletal muscle glycophenotype and identifies disease-specific changes in dystrophic muscle

Glycobiology (Oxford), 2017-12, Vol.27 (12), p.1134-1143

2017

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CD45 Signals outside of Lipid Rafts to Promote ERK Activation, Synaptic Raft Clustering, and IL-2 Production

The Journal of immunology (1950), 2005-02, Vol.174 (3), p.1479-1490

2005

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Clusters, bundles, arrays and lattices: novel mechanisms for lectin–saccharide-mediated cellular interactions

Current Opinion in Structural Biology, 2002-10, Vol.12 (5), p.616-623

2002

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Co-stimulation and counter-stimulation: lipid raft clustering controls TCR signaling and functional outcomes

Seminars in immunology, 2001-04, Vol.13 (2), p.115-128

2001

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Caveolin-1 orchestrates TCR synaptic polarity, signal specificity, and function in CD8 T cells

The Journal of immunology (1950), 2011-09, Vol.187 (6), p.2993-3002

2011

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A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

DMD genotype correlations from the Duchenne Registry: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype

Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype

Quantitative immuno-mass spectrometry imaging of skeletal muscle dystrophin

PDE5 inhibition alleviates functional muscle ischemia in boys with Duchenne muscular dystrophy

De Novo Nonsense Mutations in KAT6A, a Lysine Acetyl-Transferase Gene, Cause a Syndrome Including Microcephaly and Global Developmental Delay

A well‐tolerated core needle muscle biopsy process suitable for children and adults

Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models

The case for eteplirsen: Paving the way for precision medicine

Discs Large Homolog 1 Splice Variants Regulate p38-Dependent and -Independent Effector Functions in CD8+ T Cells

Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy

Transcriptomic analysis of paired healthy human skeletal muscles to identify modulators of disease severity in DMD

Single nuclei transcriptomics of muscle reveals intra-muscular cell dynamics linked to dystrophin loss and rescue

Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse

Emerging genetic therapies to treat Duchenne muscular dystrophy

Modeling Patient-Specific Muscular Dystrophy Phenotypes and Therapeutic Responses in Reprogrammed Myotubes Engineered on Micromolded Gelatin Hydrogels

Characterization of in vivo Dlg1 deletion on T cell development and function

FDA Approval of Eteplirsen for Muscular Dystrophy

Osteopontin promotes fibrosis in dystrophic mouse muscle by modulating immune cell subsets and intramuscular TGF-beta

Validation and Detection of Exon Skipping Boosters in DMD Patient Cell Models and mdx Mouse

Lectin-binding characterizes the healthy human skeletal muscle glycophenotype and identifies disease-specific changes in dystrophic muscle

CD45 Signals outside of Lipid Rafts to Promote ERK Activation, Synaptic Raft Clustering, and IL-2 Production

Clusters, bundles, arrays and lattices: novel mechanisms for lectin–saccharide-mediated cellular interactions

Co-stimulation and counter-stimulation: lipid raft clustering controls TCR signaling and functional outcomes

Caveolin-1 orchestrates TCR synaptic polarity, signal specificity, and function in CD8 T cells

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