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Auteur Wang D

Documents disponibles écrits par cet auteur (14)

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Human molecular genetics Real-world evidence: Risdiplam in a patient with spinal muscular atrophy type I with a novel splicing mutation and one SMN2 copy

Ma K ; Zhang K ; Chen D ; Wang C ; Abdalla M ; Zhang H ; Tian R ; Liu Y ; Song L ; Zhang X ; Liu F ; Liu G ; Wang D | 23/03/2024

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$Single-cell analysis of refractory anti-SRP necrotizing myopathy treated with anti-BCMA CAR-T cell therapy$

Article

Proceedings of the national Academy of sciences of the United States of America, 121, 6. Single-cell analysis of refractory anti-SRP necrotizing myopathy treated with anti-BCMA CAR-T cell therapy

Qin C ; Dong MH ; Zhou LQ ; Wang W ; Cai SB ; You YF ; Shang K ; Xiao J ; Wang D ; Li CR ; Zhang M ; Bu BT ; Tian DS ; Wang W | 06/02/2024

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Science (New York, N.Y.) Base editing rescue of spinal muscular atrophy in cells and in mice

Arbab M ; Matuszek Z ; Kray KM ; Du A ; Newby GA ; Blatnik AJ ; Raguram A ; Richter MF ; Zhao KT ; Levy JM ; Shen MW ; Arnold WD ; Wang D ; Xie J ; Gao G ; Burghes AHM ; Liu DR | United States | 30/03/2023

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Frontiers in neurology, 13. Efficacy and Safety of the Ketogenic Diet for Mitochondrial Disease With Epilepsy: A Prospective, Open-labeled, Controlled Study

Huang L ; Li H ; Zhong J ; Yang L ; Chen G ; Wang D ; Zheng G ; Han H ; Han X ; Long Y ; Wang X ; Liang J ; Yu M ; Shen X ; Fan M ; Fang F ; Liao J ; Sun D | 08/2022

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Article

Molecular neurobiology Treatment with IFB-088 Improves Neuropathy in CMT1A and CMT1B Mice

Bai Y ; Treins C ; Volpi VG ; Scapin C ; Ferri C ; Mastrangelo R ; Touvier T ; Florio F ; Bianchi F ; Del Carro U ; Baas FF ; Wang D ; Miniou P ; Guedat P ; Shy ME ; D'Antonio M | United States | 30/04/2022

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Article

Nature, 604, 7905. AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

Wang J ; Zhang Y ; Mendonca CA ; Yukselen O ; Muneeruddin K ; Ren L ; Liang J ; Zhou C ; Xie J ; Li J ; Jiang Z ; Kucukural A ; Shaffer SA ; Gao G ; Wang D | England | 04/2022

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Nucleic acid therapeutics, 32, 1. Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.

Servais L ; Mercuri E ; Straub V ; Guglieri M ; Seferian AM ; Scoto M ; Leone D ; Koenig E ; Khan N ; Dugar A ; Wang X ; Han B ; Wang D ; Muntoni F | 02/2022

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Nucleic acid therapeutics Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Servais L ; Mercuri E ; Straub V ; Guglieri M ; Seferian AM ; Scoto M ; Leone D ; Koenig E ; Khan N ; Dugar A ; Wang X ; Han B ; Wang D ; Muntoni F | United States | 17/11/2021

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Stem cell research, 53. Establishment of a human iPSC line XMDYYYi001-A from a patient with Becker muscular dystrophy harboring duplications of exons 2-19 in dystrophin gene

Wang D ; Lin J ; Jin M ; Wang N | England | 05/2021

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Journal of medical genetics Single-molecule optical mapping enables quantitative measurement of D4Z4 repeats in facioscapulohumeral muscular dystrophy (FSHD)

Dai Y, Auteur ; Li P ; Wang Z ; Liang F ; Yang F ; Fang L ; Huang Y ; Huang S ; Zhou J ; Wang D ; Cui L ; Wang K | England | 09/2019

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Article

Neuromuscular disorders : NMD, 29, n5. Recessive mutations in proximal I-band of TTN gene cause severe congenital multi-minicore disease without cardiac involvement.

Ge L, Auteur ; Fu X ; Zhang W ; Wang D ; Wang Z ; Yuan Y ; Nonaka I ; Xiong H | England | 05/2019

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Article

Cell stem cell, 18, 4. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

Young CS, Auteur ; Hicks MR ; Ermolova NV ; Nakano H ; Jan M ; Younesi S ; Karumbayaram S ; Kumagai-Cresse C ; Wang D ; Zack JA ; Kohn DB ; Nakano A ; Nelson SF ; Miceli MC ; Spencer MJ ; Pyle AD | 2016

Comment in: Imprecision Medicine: A One-Size-Fits-Many Approach for Muscle Dystrophy. [Cell Stem Cell. 2016]

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Article

Neurology, 85, 10. Quality improvement in neurology: Muscular dystrophy quality measures.

Narayanaswami P ; Dubinsky R ; Wang D ; Gjorvad G ; David W ; Finder J ; Smith B ; Cheng J ; Shapiro F ; Mellion M ; Spurney C ; Wolff J ; England J | 08/09/2015

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Article

Human molecular genetics, 23, 10. Role of toll like receptors in the pathogenesis of dystrophin-deficient skeletal and heart muscle

Henriques-Pons A ; Yu Q ; Rayavarapu S ; Cohen TV ; Ampong B ; Cha HJ ; Jahnke V ; VanderMeulen J ; Wang D ; Wayne J ; Kandimalla ER ; Agrawal S ; Spurney CF ; Nagaraju K | 2014

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Auteur Wang D

Documents disponibles écrits par cet auteur (14)

Human molecular genetics Real-world evidence: Risdiplam in a patient with spinal muscular atrophy type I with a novel splicing mutation and one SMN2 copy

Proceedings of the national Academy of sciences of the United States of America, 121, 6. Single-cell analysis of refractory anti-SRP necrotizing myopathy treated with anti-BCMA CAR-T cell therapy

Science (New York, N.Y.) Base editing rescue of spinal muscular atrophy in cells and in mice

Frontiers in neurology, 13. Efficacy and Safety of the Ketogenic Diet for Mitochondrial Disease With Epilepsy: A Prospective, Open-labeled, Controlled Study

Molecular neurobiology Treatment with IFB-088 Improves Neuropathy in CMT1A and CMT1B Mice

Nature, 604, 7905. AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

Nucleic acid therapeutics, 32, 1. Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.

Nucleic acid therapeutics Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Stem cell research, 53. Establishment of a human iPSC line XMDYYYi001-A from a patient with Becker muscular dystrophy harboring duplications of exons 2-19 in dystrophin gene

Journal of medical genetics Single-molecule optical mapping enables quantitative measurement of D4Z4 repeats in facioscapulohumeral muscular dystrophy (FSHD)

Neuromuscular disorders : NMD, 29, n5. Recessive mutations in proximal I-band of TTN gene cause severe congenital multi-minicore disease without cardiac involvement.

Cell stem cell, 18, 4. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

Neurology, 85, 10. Quality improvement in neurology: Muscular dystrophy quality measures.

Human molecular genetics, 23, 10. Role of toll like receptors in the pathogenesis of dystrophin-deficient skeletal and heart muscle