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Precise therapeutic gene correction by a simple nuclease-induced double-stranded break

Nature (London), 2019-04, Vol.568 (7753), p.561-565

2019

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Phosphoinositide 3-Kinase and Akt Are Essential for Sonic Hedgehog Signaling

Proceedings of the National Academy of Sciences - PNAS, 2006-03, Vol.103 (12), p.4505-4510

2006

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SULF1 and SULF2 regulate heparan sulfate-mediated GDNF signaling for esophageal innervation

Development (Cambridge), 2007-09, Vol.134 (18), p.3327-3338

2007

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Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics

Molecular therapy, 2016-08, Vol.24 (8), p.1405-1411

2016

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SMCHD1 activates the expression of genes required for the expansion of human myoblasts

Nucleic acids research, 2024-07, Vol.52 (16), p.9450-9462

2024

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CRISPR/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion

Genome Biology, 2017-06, Vol.18 (1), p.108-8, Article 108

2017

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Telomere position effect regulates DUX4 in human facioscapulohumeral muscular dystrophy

Nature structural & molecular biology, 2013-06, Vol.20 (6), p.671-678

2013

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p38 MAPKs - roles in skeletal muscle physiology, disease mechanisms, and as potential therapeutic targets

JCI insight, 2021-06, Vol.6 (12)

2021

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Increasing intracellular dNTP levels improves prime editing efficiency

Nature biotechnology, 2024-09

2024

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Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism

Cell death & disease, 2023-11, Vol.14 (11), p.749-749, Article 749

2023

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QSulf1 Remodels the 6-O Sulfation States of Cell Surface Heparan Sulfate Proteoglycans to Promote Wnt Signaling

The Journal of cell biology, 2003-07, Vol.162 (2), p.341-351

2003

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Transcriptional profiling in facioscapulohumeral muscular dystrophy to identify candidate biomarkers

Proceedings of the National Academy of Sciences - PNAS, 2012-10, Vol.109 (40), p.16234-16239

2012

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Impaired mitochondrial oxidative metabolism in skeletal progenitor cells leads to musculoskeletal disintegration

Nature communications, 2022-11, Vol.13 (1), p.6869-6869, Article 6869

2022

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Pax3 synergizes with Gli2 and Zic1 in transactivating the Myf5 epaxial somite enhancer

Developmental biology, 2013-11, Vol.383 (1), p.7-14

2013

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MYOGENIC REGULATORY FACTORS AND THE SPECIFICATION OF MUSCLE PROGENITORS IN VERTEBRATE EMBRYOS

Annual review of cell and developmental biology, 2002-01, Vol.18 (1), p.747-783

2002

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Facioscapulohumeral muscular dystrophy family studies of DUX4 expression: evidence for disease modifiers and a quantitative model of pathogenesis

Human molecular genetics, 2012-10, Vol.21 (20), p.4419-4430

2012

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Outcome Measures in Facioscapulohumeral Muscular Dystrophy Clinical Trials

Cells (Basel, Switzerland), 2022-02, Vol.11 (4), p.687

2022

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Substrate Specificity and Domain Functions of Extracellular Heparan Sulfate 6-O-Endosulfatases, QSulf1 and QSulf2

The Journal of biological chemistry, 2006-02, Vol.281 (8), p.4969-4976

2006

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Regulation of Wnt Signaling and Embryo Patterning by an Extracellular Sulfatase

Science (American Association for the Advancement of Science), 2001-08, Vol.293 (5535), p.1663-1666

2001

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DUX4 expression activates JNK and p38 MAP kinases in myoblasts

Disease models & mechanisms, 2022-11, Vol.15 (11)

2022

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Facioscapulohumeral muscular dystrophy family studies of DUX4 expression: evidence for disease modifiers and a quantitative model of pathogenesis

Human molecular genetics, 2012-10, Vol.21 (20), p.4419-4430

2012

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iMyoblasts for ex vivo and in vivo investigations of human myogenesis and disease modeling

eLife, 2022-01, Vol.11

2022

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Efficient Homology-Directed Repair with Circular Single-Stranded DNA Donors

CRISPR journal, 2022-10, Vol.5 (5), p.685-701

2022

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QSulf1, a Heparan Sulfate 6-O-Endosulfatase, Inhibits Fibroblast Growth Factor Signaling in Mesoderm Induction and Angiogenesis

Proceedings of the National Academy of Sciences - PNAS, 2004-04, Vol.101 (14), p.4833-4838

2004

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Establishment of clonal myogenic cell lines from severely affected dystrophic muscles - CDK4 maintains the myogenic population

Skeletal muscle, 2011-03, Vol.1 (1), p.12-12, Article 12

2011

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Precise therapeutic gene correction by a simple nuclease-induced double-stranded break

Phosphoinositide 3-Kinase and Akt Are Essential for Sonic Hedgehog Signaling

SULF1 and SULF2 regulate heparan sulfate-mediated GDNF signaling for esophageal innervation

Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics

SMCHD1 activates the expression of genes required for the expansion of human myoblasts

CRISPR/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion

Telomere position effect regulates DUX4 in human facioscapulohumeral muscular dystrophy

p38 MAPKs - roles in skeletal muscle physiology, disease mechanisms, and as potential therapeutic targets

Increasing intracellular dNTP levels improves prime editing efficiency

Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism

QSulf1 Remodels the 6-O Sulfation States of Cell Surface Heparan Sulfate Proteoglycans to Promote Wnt Signaling

Transcriptional profiling in facioscapulohumeral muscular dystrophy to identify candidate biomarkers

Impaired mitochondrial oxidative metabolism in skeletal progenitor cells leads to musculoskeletal disintegration

Pax3 synergizes with Gli2 and Zic1 in transactivating the Myf5 epaxial somite enhancer

MYOGENIC REGULATORY FACTORS AND THE SPECIFICATION OF MUSCLE PROGENITORS IN VERTEBRATE EMBRYOS

Facioscapulohumeral muscular dystrophy family studies of DUX4 expression: evidence for disease modifiers and a quantitative model of pathogenesis

Outcome Measures in Facioscapulohumeral Muscular Dystrophy Clinical Trials

Substrate Specificity and Domain Functions of Extracellular Heparan Sulfate 6-O-Endosulfatases, QSulf1 and QSulf2

Regulation of Wnt Signaling and Embryo Patterning by an Extracellular Sulfatase

DUX4 expression activates JNK and p38 MAP kinases in myoblasts

Facioscapulohumeral muscular dystrophy family studies of DUX4 expression: evidence for disease modifiers and a quantitative model of pathogenesis

iMyoblasts for ex vivo and in vivo investigations of human myogenesis and disease modeling

Efficient Homology-Directed Repair with Circular Single-Stranded DNA Donors

QSulf1, a Heparan Sulfate 6-O-Endosulfatase, Inhibits Fibroblast Growth Factor Signaling in Mesoderm Induction and Angiogenesis

Establishment of clonal myogenic cell lines from severely affected dystrophic muscles - CDK4 maintains the myogenic population

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