Research graph
References from Gene therapy in Duchenne muscular dystrophy (DMD). Local targets link to admitted publications; unresolved targets remain external evidence.
Generation and characterization of transgenic mice with the full-length human DMD gene
10.1074/jbc.m709410200 · 2008 · External reference
The 10th oligonucleotide therapy approved: golodirsen for duchenne muscular dystrophy
10.1089/nat.2020.0845 · 2020 · External reference
A sequel to the eteplirsen saga: eteplirsen is approved in the United States but was not approved in Europe
10.1089/nat.2018.0756 · 2019 · External reference
FDA Approves eteplirsen for Duchenne muscular dystrophy: the next chapter in the eteplirsen saga
10.1089/nat.2016.0657 · 2017 · External reference
Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading‐frame rule
10.1002/mus.20586 · 2006 · External reference
Unresolved reference
2026 · External reference
The structural and functional diversity of dystrophin
10.1038/ng0493-283 · 1993 · External reference
A voyage on the role of nuclear factor kappa B (NF-kB) signaling pathway in duchenne muscular dystrophy: an inherited muscle disorder
2024 · External reference
Absence of dystrophin disrupts skeletal muscle signaling: roles of Ca2+, reactive oxygen species, and nitric oxide in the development of muscular dystrophy
10.1152/physrev.00007.2015 · 2015 · External reference
A cluster of basic repeats in the dystrophin rod domain binds F-actin through an electrostatic interaction
10.1074/jbc.273.43.28419 · 1998 · External reference
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
10.1126/science.aau1549 · 2018 · External reference
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
10.1126/scitranslmed.aan8081 · 2017 · External reference
Exon skipping quantification by quantitative reverse-transcription polymerase chain reaction in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen
10.1089/hgtb.2012.117 · 2012 · External reference
Targeted disruption of Exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in duchenne muscular dystrophy
10.1006/bbrc.1997.7328 · 1997 · External reference
A new immuno-, dystrophin-deficient model, the NSG-mdx4Cv mouse, provides evidence for functional improvement following allogeneic satellite cell transplantation
10.1002/stem.1402 · 2013 · External reference
Clinical development on the frontier: gene therapy for duchenne muscular dystrophy
10.1080/14712598.2020.1725469 · 2020 · External reference
Muscular dystrophy in the Japanese Spitz: an inversion disrupts the DMD and RPGR genes
10.1111/age.12266 · 2015 · External reference
Caregiver-reported patient experiences with duchenne muscular dystrophy: qualitative in-trial interviews 1 year after delandistrogene moxeparvovec in the pivotal EMBARK trial
10.1007/s40120-025-00842-7 · 2026 · External reference
Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy
10.1371/journal.pone.0283669 · 2023 · External reference
Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy
10.1016/j.xcrm.2025.102037 · 2025 · External reference
Long-term survival and myocardial function following systemic delivery of delandistrogene moxeparvovec in DMD MDX Rats
10.1089/hum.2024.013 · 2024 · External reference
The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins
10.1371/journal.pgen.1000958 · 2010 · External reference
Is it time for genetic modifiers to predict prognosis in Duchenne muscular dystrophy?
10.1038/s41582-023-00823-0 · 2023 · External reference
Activin A antagonism with follistatin reduces kidney fibrosis, injury, and cellular senescence-associated inflammation in murine diabetic kidney disease
10.34067/kid.0000000776 · 2025 · External reference
Unresolved reference
2019 · External reference
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
10.1016/s1474-4422(18)30024-3 · 2018 · External reference
The TREAT-NMD DMD global database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
10.1002/humu.22758 · 2015 · External reference
The TREAT-NMD DMD global database: analysis of more than 7,000 duchenne muscular dystrophy mutations
10.1002/humu.22758 · 2015 · External reference
N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy
10.1186/s13395-023-00334-y · 2024 · External reference
Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy
10.1016/0887-8994(95)00251-0 · 1996 · External reference
Duchenne muscular dystrophy: deficiency of dystrophin at the muscle cell surface
10.1016/0092-8674(88)90065-7 · 1988 · External reference
Cognitive impairment in Duchenne muscular dystrophy
10.1016/0960-8966(94)90072-8 · 1994 · External reference
Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy
10.1212/wnl.39.4.475 · 1989 · External reference
Noncoding RNAs in Duchenne and Becker muscular dystrophies: role in pathogenesis and future prognostic and therapeutic perspectives
10.1007/s00018-020-03537-4 · 2020 · External reference
The role of mitochondria in duchenne muscular dystrophy
2021 · External reference
X chromosome-linked muscular dystrophy (mdx) in the mouse
10.1073/pnas.81.4.1189 · 1984 · External reference
Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings
10.4155/cli.11.113 · 2011 · External reference
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial
10.1038/s41591-025-03750-3 · 2025 · External reference
Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy
10.1016/j.ymthe.2025.06.032 · 2025 · External reference
Current clinical applications of AAV-mediated gene therapy
10.1016/j.ymthe.2025.04.045 · 2025 · External reference
Progress toward gene therapy for duchenne muscular dystrophy
10.1016/j.ymthe.2017.02.019 · 2017 · External reference
Dystrophin-deficient mdx mice display a reduced life span and are susceptible to spontaneous rhabdomyosarcoma
10.1096/fj.06-7353com · 2007 · External reference
Recovery of induced mutations for X chromosome-linked muscular dystrophy in mice
10.1073/pnas.86.4.1292 · 1989 · External reference
Predictors of death in adults with duchenne muscular dystrophy–associated cardiomyopathy
10.1161/jaha.117.006340 · 2017 · External reference
Correction of muscular dystrophies by CRISPR gene editing
10.1172/jci136873 · 2020 · External reference
CRISPR-Editing therapy for Duchenne muscular dystrophy
10.1089/hum.2023.053 · 2023 · External reference
Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9
10.1093/hmg/ddv120 · 2015 · External reference
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery
10.1038/mt.2013.244 · 2014 · External reference
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates
10.1038/mt.2013.246 · 2014 · External reference
Delivery of genetic medicines for muscular dystrophies
10.1016/j.xcrm.2024.101885 · 2025 · External reference
AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects
10.1038/s41434-025-00561-6 · 2025 · External reference
Efficacy and safety of viltolarsen in boys with Duchenne muscular dystrophy: results from the phase 2, open-label, 4-year extension study
10.3233/jnd-221656 · 2023 · External reference
Safety, tolerability, and efficacy of viltolarsen in boys with duchenne muscular dystrophy amenable to exon 53 skipping: a phase 2 randomized clinical trial
10.1001/jamaneurol.2020.1264 · 2020 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Unresolved reference
2026 · External reference
Delays in diagnosis of Duchenne muscular dystrophy: an evaluation of genotypic and sociodemographic factors
10.1002/mus.26720 · 2020 · External reference
Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis
10.1186/s13023-020-01430-8 · 2020 · External reference
Nox4—RyR1—Nox2: regulators of micro-domain signaling in skeletal muscle
10.1016/j.redox.2020.101557 · 2020 · External reference
Evolving therapeutic options for the treatment of Duchenne muscular dystrophy
10.1007/s13311-023-01423-y · 2023 · External reference
The frequency of revertants in mdx mouse genetic models for duchenne muscular dystrophy
10.1203/00006450-199207000-00025 · 1992 · External reference
Neurodevelopmental, behavioral, and emotional symptoms common in Duchenne muscular dystrophy
10.1002/mus.26803 · 2020 · External reference
Survival in Duchenne muscular dystrophy in Australia: a 50 year retrospective cohort study
10.1016/j.lanwpc.2025.101568 · 2025 · External reference
Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy
10.1016/s0092-8674(00)80532-2 · 1997 · External reference
Micro-dystrophin gene therapy goes systemic in Duchenne muscular dystrophy patients
10.1089/hum.2018.012 · 2018 · External reference
Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy
10.1016/j.ymthe.2018.07.011 · 2018 · External reference
Duchenne muscular dystrophy
10.1038/s41572-021-00248-3 · 2021 · External reference
Full-length dystrophin gene therapy for Duchenne muscular dystrophy
10.1016/j.ymthe.2024.07.026 · 2024 · External reference
Functional role of store‐operated and stretch‐activated channels in murine adult skeletal muscle fibres
10.1113/jphysiol.2006.115154 · 2006 · External reference
Unresolved reference
2024 · External reference
Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation
10.1016/s0960-8966(02)00140-2 · 2002 · External reference
A dystrophin exon-52 deleted miniature pig model of duchenne muscular dystrophy and evaluation of exon skipping
10.3390/ijms222313065 · 2021 · External reference
Next-generation sequencing in neuromuscular diseases
10.1097/wco.0000000000000374 · 2016 · External reference
CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene
10.1242/dmm.037655 · 2019 · External reference
Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo
10.1128/jvi.77.13.7689-7695.2003 · 2003 · External reference
Population frequencies of inherited neuromuscular diseases—a world survey
10.1016/0960-8966(91)90039-u · 1991 · External reference
Very mild muscular dystrophy associated with the deletion of 46% of dystrophin
10.1038/343180a0 · 1990 · External reference
CRISPR Therapeutics for Duchenne muscular dystrophy
10.3390/ijms23031832 · 2022 · External reference
Dystrophin, its interactions with other proteins, and implications for muscular dystrophy
10.1016/j.bbadis.2006.05.010 · 2007 · External reference
Duchenne and Becker muscular dystrophy: cellular mechanisms, image analysis, and computational models: a review
10.1002/cm.21826 · 2024 · External reference
T and B lymphocyte depletion has a marked effect on the fibrosis of dystrophic skeletal muscles in the scid/mdx mouse
10.1002/path.2213 · 2007 · External reference
Prognostic indicators of disease progression in Duchenne muscular dystrophy: a literature review and evidence synthesis
10.1371/journal.pone.0265879 · 2022 · External reference
DMD Trp3X nonsense mutation associated with a founder effect in North American families with mild Becker muscular dystrophy
10.1016/j.nmd.2009.08.010 · 2009 · External reference
Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort
10.1002/humu.21114 · 2009 · External reference
A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2
10.1016/j.omtm.2022.08.009 · 2022 · External reference
Biomarkers in Duchenne muscular dystrophy: current status and future directions
10.3233/jnd-221666 · 2023 · External reference
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
10.1212/wnl.0000000000009233 · 2020 · External reference
Critical evaluation of cytosolic calcium determination in resting muscle fibres from normal and dystrophic (mdx) mice
10.1016/0143-4160(93)90006-r · 1993 · External reference
In situ measurements of calpain activity in isolated muscle fibres from normal and dystrophin‐lacking mdx mice
10.1113/jphysiol.2007.132191 · 2007 · External reference
European Society of Gene & Cell Therapy Spring School 2025
10.1089/hum.2025.088 · 2025 · External reference
The dystrophin complex: structure, function, and implications for therapy
10.1002/j.2040-4603.2015.tb00638.x · 2015 · External reference
The complex landscape of DMD mutations: moving towards personalized medicine
10.3389/fgene.2024.1360224 · 2024 · External reference
A patient-centered qualitative evaluation of meaningful change on the NSAA and PUL in Duchenne Muscular Dystrophy
10.3389/fneur.2025.1509174 · 2025 · External reference
Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy
10.1016/s0092-8674(00)80533-4 · 1997 · External reference
Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy
10.1212/wnl.0000000000003217 · 2016 · External reference
DMD exon 1 truncating point mutations: amelioration of phenotype by alternative translation initiation in exon 6
10.1002/humu.20913 · 2009 · External reference
The D2.mdx mouse as a preclinical model of the skeletal muscle pathology associated with Duchenne muscular dystrophy
10.1038/s41598-020-70987-y · 2020 · External reference
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy
10.1038/nm0302-253 · 2002 · External reference
Disease progression modeling of the North Star Ambulatory Assessment for Duchenne Muscular Dystrophy
10.1002/psp4.12921 · 2023 · External reference
Gene therapy
10.1056/nejmra1706910 · 2019 · External reference
Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN
10.1089/hum.2018.015 · 2018 · External reference
Dystrophin: the protein product of the duchenne muscular dystrophy locus
10.1016/0092-8674(87)90579-4 · 1987 · External reference
Dystrophin abnormalities in Duchenne/Becker muscular dystrophy
10.1016/0896-6273(89)90226-2 · 1989 · External reference
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury
10.1016/j.ymthe.2024.02.002 · 2024 · External reference
Sequence specificity of aminoglycoside-induced stop codon readthrough: potential implications for treatment of Duchenne muscular dystrophy
10.1002/1531-8249(200008)48:2<164::aid-ana5>3.0.co;2-b · 2000 · External reference
Multiplex ligation-dependent probe amplification identification of deletions and duplications of the Duchenne muscular dystrophy gene in taiwanese subjects
10.1016/s0929-6646(09)60318-1 · 2007 · External reference
Differential expression of dystrophin isoforms in strains of mdx mice with different mutations
10.1093/hmg/5.8.1149 · 1996 · External reference
Muscle phenotype of a rat model of Duchenne muscular dystrophy
10.1002/mus.27061 · 2020 · External reference
Incidence of Duchenne muscular dystrophy in the modern era; an Australian study
10.1038/s41431-022-01138-2 · 2022 · External reference
Vamorolone: first approval
10.1007/s40265-023-01986-2 · 2024 · External reference
Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice
10.1038/s41467-021-26714-w · 2021 · External reference
Expanding the potential of gene therapy for duchenne muscular dystrophy
10.2174/0113816128386290250507101412 · 2026 · External reference
Highly efficient RNA-guided base editing in mouse embryos
10.1038/nbt.3816 · 2017 · External reference
Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle
10.1093/hmg/ddt287 · 2013 · External reference
Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals
10.1016/0092-8674(87)90504-6 · 1987 · External reference
Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using campylobacter jejuni Cas9
10.1016/j.ymthe.2018.03.018 · 2018 · External reference
Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies
10.1007/s00335-011-9382-y · 2012 · External reference
Follistatin gene delivery enhances muscle growth and strength in nonhuman primates
10.1126/scitranslmed.3000112 · 2009 · External reference
Neighboring-nucleotide effects on the rates of germ-line single-base- pair substitution in human genes
10.1086/301965 · 1998 · External reference
William Allan Award address. Cloning of the DMD gene
10.1086/428143 · 2005 · External reference
Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy
10.1172/jci36612 · 2009 · External reference
Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy
10.1371/journal.pone.0110371 · 2014 · External reference
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
10.1038/ncomms16105 · 2017 · External reference
Dystrophin as a molecular shock absorber
10.1021/acsnano.8b05721 · 2018 · External reference
An updated analysis of exon-skipping applicability for Duchenne muscular dystrophy using the UMD-DMD database
10.3390/genes15111489 · 2024 · External reference
The hidden disease: delayed diagnosis in Duchenne muscular dystrophy and co-occurring conditions
10.1097/dbp.0000000000001105 · 2022 · External reference
Clinical and genetic characterization of female dystrophinopathy
10.3988/jcn.2015.11.3.248 · 2015 · External reference
Death after high-dose rAAV9 gene therapy in a patient with Duchenne’s muscular dystrophy
10.1056/nejmoa2307798 · 2023 · External reference
Loop engineering improves prime editing efficiency
10.1016/j.omtn.2025.102764 · 2025 · External reference
CRISPR-Cas9-mediated homology-directed repair for precise gene editing
10.1016/j.omtn.2024.102344 · 2024 · External reference
Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA
10.1126/science.1254445 · 2014 · External reference
Cardiac MRI biomarkers for Duchenne muscular dystrophy
10.2217/bmm-2018-0125 · 2018 · External reference
Genetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice
10.1186/1471-2199-10-15 · 2009 · External reference
A population-based study of dystrophin mutations in Canada
10.1017/s0317167100011896 · 2011 · External reference
Cas9 as a versatile tool for engineering biology
10.1038/nmeth.2649 · 2013 · External reference
Glucocorticoid corticosteroids for Duchenne muscular dystrophy
2008 · External reference
Abnormal calcium handling in duchenne muscular dystrophy: mechanisms and potential therapies
10.3389/fphys.2021.647010 · 2021 · External reference
Extensive striated muscle damage in a rat model of Duchenne muscular dystrophy with Dmd exons 10–17 duplication
10.1186/s13395-025-00386-2 · 2025 · External reference
Therapeutic strategies for dystrophin replacement in Duchenne muscular dystrophy
2022 · External reference
Profiles of neuromuscular diseases: duchenne muscular dystrophy
10.1097/00002060-199509001-00004 · 1995 · External reference
Caregiver global impression observations from EMBARK: a phase 3 study evaluating delandistrogene moxeparvovec in ambulatory patients with Duchenne muscular dystrophy
10.1007/s40120-024-00685-8 · 2025 · External reference
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study
10.1002/mus.23905 · 2013 · External reference
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
10.1242/dmm.018424 · 2015 · External reference
CRISPR activation: identifying and using novel genes for plant disease resistance breeding
10.3389/fgeed.2025.1596600 · 2025 · External reference
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
10.1038/s41591-024-03304-z · 2025 · External reference
Gene delivery for Limb-Girdle Muscular dystrophy type 2D by isolated limb infusion
10.1089/hum.2019.006 · 2019 · External reference
Two-year outcomes following delandistrogene moxeparvovec treatment in ambulatory patients with duchenne muscular dystrophy: phase 3 EMBARK trial
10.1007/s40120-025-00879-8 · 2026 · External reference
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
10.1038/s41591-024-03304-z · 2025 · External reference
Gene therapy for muscular dystrophy: lessons learned and path forward
10.1016/j.neulet.2012.04.078 · 2012 · External reference
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: a phase 1/2a nonrandomized trial
10.1002/mus.27955 · 2024 · External reference
Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial
10.1001/jamaneurol.2020.1484 · 2020 · External reference
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy
10.1038/mt.2014.200 · 2015 · External reference
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy
10.3389/fcell.2023.1167762 · 2023 · External reference
Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim analysis
10.1007/s00415-023-11687-1 · 2023 · External reference
Steroids in duchenne muscular dystrophy—deflazacort trial
10.1016/0960-8966(91)90099-e · 1991 · External reference
Clinical management of Duchenne muscular dystrophy: the state of the art
10.1007/s10072-018-3555-3 · 2018 · External reference
Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis
10.1073/pnas.0609411103 · 2006 · External reference
Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing
10.1016/j.ymthe.2020.05.024 · 2020 · External reference
CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
10.1126/sciadv.aav4324 · 2019 · External reference
An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
10.1016/0888-7543(88)90113-9 · 1988 · External reference
Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene
10.1038/323646a0 · 1986 · External reference
Dystrophin restoration after adeno-associated virus U7–mediated Dmd exon skipping is modulated by muscular exercise in the severe D2-Mdx Duchenne muscular dystrophy murine model
10.1016/j.ajpath.2022.07.016 · 2022 · External reference
Role of telomere dysfunction in cardiac failure in Duchenne muscular dystrophy
10.1038/ncb2790 · 2013 · External reference
FDA Approves first gene therapy for Duchenne muscular dystrophy, despite internal objections
10.1038/d41573-023-00103-y · 2023 · External reference
Transduction efficiency of adeno-associated virus serotypes after local injection in mouse and human skeletal muscle
10.1089/hum.2019.173 · 2020 · External reference
MRI Vastus lateralis fat fraction predicts loss of ambulation in Duchenne muscular dystrophy
10.1212/wnl.0000000000008939 · 2020 · External reference
Generation of muscular dystrophy model rats with a CRISPR/Cas system
10.1038/srep05635 · 2014 · External reference
New selective androgen receptor modulator TEI-SARM2 improves muscle function in a Duchenne muscular dystrophy rat model
10.1093/hmg/ddaf028 · 2025 · External reference
Unresolved reference
2026 · External reference
Engineered pegRNAs improve prime editing efficiency
10.1038/s41587-021-01039-7 · 2022 · External reference
A defect in dystrophin causes a novel porcine stress syndrome
10.1186/1471-2164-13-233 · 2012 · External reference
Dystrophin and muscular dystrophy: past, present, and future
10.1006/mgme.2001.3220 · 2001 · External reference
Generation of a dystrophin mutant in dog by nuclear transfer using CRISPR/Cas9-mediated somatic cells: a preliminary study
10.3390/ijms23052898 · 2022 · External reference
Consensus on the diagnosis, treatment and follow-up of patients with Duchenne muscular dystrophy
10.1016/j.nrleng.2018.01.001 · 2019 · External reference
Delivery challenges for CRISPR-Cas9 genome editing for Duchenne muscular dystrophy
10.1063/5.0131452 · 2023 · External reference
Deciphering the molecular mechanism of stop codon readthrough
10.1111/brv.12657 · 2021 · External reference
Boosting plant genome editing with a versatile CRISPR-Combo system
10.1038/s41477-022-01151-9 · 2022 · External reference
Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes
10.1371/journal.pone.0108205 · 2014 · External reference
Food and drug administration lifts clinical hold on pfizer duchenne muscular dystrophy gene therapy linked to patient death
10.1089/hum.2022.29211.bfs · 2022 · External reference
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s death fails phase III Trial
10.1089/hum.2024.38567.bfs · 2024 · External reference
Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy
10.1164/ajrccm.164.12.2103052 · 2001 · External reference
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD
10.1038/mt.2011.59 · 2011 · External reference
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy
10.1016/j.ymthe.2021.09.003 · 2021 · External reference
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy
10.1038/s41598-024-84077-w · 2025 · External reference
Development of novel micro-dystrophins with enhanced functionality
10.1016/j.ymthe.2019.01.002 · 2019 · External reference
Base editing: precision chemistry on the genome and transcriptome of living cells
10.1038/s41576-018-0059-1 · 2018 · External reference
The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
2016 · External reference
Systemic gene transfer with rAAVrh74. MHCK7. SGCB Increased β-sarcoglycan expression in patients with Limb Girdle muscular dystrophy type 2E
2019 · External reference
Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease
10.1002/mus.21244 · 2009 · External reference
Severe muscular dystrophy in mice that lack dystrophin and α7 integrin
10.1242/jcs.02952 · 2006 · External reference
Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx/mTR mice
10.1016/j.cell.2010.11.039 · 2010 · External reference
Global prevalence of Duchenne and Becker muscular dystrophy: a systematic review and meta-analysis
2022 · External reference
Global prevalence of Duchenne and Becker muscular dystrophy: a systematic review and meta-analysis
2022 · External reference
Thrombotic microangiopathy as an emerging complication of viral vector–based gene therapy
10.1016/j.ekir.2024.04.024 · 2024 · External reference
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
10.1089/nat.2021.0043 · 2022 · External reference
An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy
10.1016/0888-7543(92)90210-j · 1992 · External reference
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European Society of Gene & Cell Therapy Spring School 2025
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Follistatin gene delivery enhances muscle growth and strength in nonhuman primates
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Severe muscular dystrophy in mice that lack dystrophin and α7 integrin
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