Abstract
Arezou Azizsoltani, Zeinab Ghesmati, Elnaz Rezaei
Abstract
Authors
Institutions
Provenance
crossref
Confidence 100%
ror
Confidence 99%
No local reference links have been materialized yet.
No local citing links have been materialized yet.
Generation and characterization of transgenic mice with the full-length human DMD gene
10.1074/jbc.m709410200 · 2008
The 10th oligonucleotide therapy approved: golodirsen for duchenne muscular dystrophy
10.1089/nat.2020.0845 · 2020
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
FDA Approves eteplirsen for Duchenne muscular dystrophy: the next chapter in the eteplirsen saga
10.1089/nat.2016.0657 · 2017
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
Unresolved referenced work
2026
The structural and functional diversity of dystrophin
10.1038/ng0493-283 · 1993
A voyage on the role of nuclear factor kappa B (NF-kB) signaling pathway in duchenne muscular dystrophy: an inherited muscle disorder
2024
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
openalex
Confidence 95%
datacite
Confidence 0%
A cluster of basic repeats in the dystrophin rod domain binds F-actin through an electrostatic interaction
10.1074/jbc.273.43.28419 · 1998
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
10.1126/science.aau1549 · 2018
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
10.1126/scitranslmed.aan8081 · 2017
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
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
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
Clinical development on the frontier: gene therapy for duchenne muscular dystrophy
10.1080/14712598.2020.1725469 · 2020
Muscular dystrophy in the Japanese Spitz: an inversion disrupts the DMD and RPGR genes
10.1111/age.12266 · 2015
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
Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy
10.1371/journal.pone.0283669 · 2023
Single-cut gene therapy in a one-step generated rhesus monkey model of Duchenne muscular dystrophy
10.1016/j.xcrm.2025.102037 · 2025
Long-term survival and myocardial function following systemic delivery of delandistrogene moxeparvovec in DMD MDX Rats
10.1089/hum.2024.013 · 2024
The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins
10.1371/journal.pgen.1000958 · 2010
Is it time for genetic modifiers to predict prognosis in Duchenne muscular dystrophy?
10.1038/s41582-023-00823-0 · 2023
Activin A antagonism with follistatin reduces kidney fibrosis, injury, and cellular senescence-associated inflammation in murine diabetic kidney disease
10.34067/kid.0000000776 · 2025
Unresolved referenced work
2019
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
The TREAT-NMD DMD global database: analysis of more than 7,000 Duchenne muscular dystrophy mutations
10.1002/humu.22758 · 2015
The TREAT-NMD DMD global database: analysis of more than 7,000 duchenne muscular dystrophy mutations
10.1002/humu.22758 · 2015
N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy
10.1186/s13395-023-00334-y · 2024
Skeletal, cardiac, and smooth muscle failure in Duchenne muscular dystrophy
10.1016/0887-8994(95)00251-0 · 1996
Duchenne muscular dystrophy: deficiency of dystrophin at the muscle cell surface
10.1016/0092-8674(88)90065-7 · 1988
Cognitive impairment in Duchenne muscular dystrophy
10.1016/0960-8966(94)90072-8 · 1994
Duchenne muscular dystrophy: patterns of clinical progression and effects of supportive therapy
10.1212/wnl.39.4.475 · 1989
Noncoding RNAs in Duchenne and Becker muscular dystrophies: role in pathogenesis and future prognostic and therapeutic perspectives
10.1007/s00018-020-03537-4 · 2020
The role of mitochondria in duchenne muscular dystrophy
2021
X chromosome-linked muscular dystrophy (mdx) in the mouse
10.1073/pnas.81.4.1189 · 1984
Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings
10.4155/cli.11.113 · 2011
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial
10.1038/s41591-025-03750-3 · 2025
Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy
10.1016/j.ymthe.2025.06.032 · 2025
Current clinical applications of AAV-mediated gene therapy
10.1016/j.ymthe.2025.04.045 · 2025
Comparison of serum rAAV serotype-specific antibodies in patients with Duchenne muscular dystrophy, becker muscular dystrophy, inclusion body myositis, or GNE myopathy
10.1089/hum.2016.141 · doi-reference
Analysis of AAV serotypes 1–9 mediated gene expression and tropism in mice after systemic injection
10.1038/mt.2008.76 · doi-reference
Dystrophin contains multiple independent membrane-binding domains
10.1093/hmg/ddw210 · doi-reference
Delandistrogene moxeparvovec gene therapy in ambulatory patients (aged ≥4 to ESCAPED_CHARACTER_LESS_THAN8 Years) with Duchenne muscular dystrophy: 1-Year interim results from study SRP-9001-103 (ENDEAVOR
10.1002/ana.26755 · doi-reference
Disrupted calcium homeostasis in duchenne muscular dystrophy: a common mechanism behind diverse consequences
10.3390/ijms222011040 · doi-reference
Humanizing the mdx mouse model of DMD: the long and the short of it
10.1038/s41536-018-0045-4 · doi-reference
Genetic modulation of RNA splicing with a CRISPR-guided cytidine deaminase
10.1016/j.molcel.2018.09.002 · doi-reference
Creation of a novel humanized dystrophic mouse model of Duchenne muscular dystrophy and application of a CRISPR/Cas9 gene editing therapy
10.3233/jnd-170218 · doi-reference
CRISPR/Cas9-based gene activation and base editing in Populus
10.1093/hr/uhad085 · doi-reference
Overexpression of Galgt2 reduces dystrophic pathology in the skeletal muscles of alpha sarcoglycan-deficient mice
10.2353/ajpath.2009.080967 · doi-reference
Efficient base editing for multiple genes and loci in pigs using base editors
10.1038/s41467-019-10421-8 · doi-reference
The role of the dystrophin glycoprotein complex in muscle cell mechanotransduction
10.1038/s42003-022-03980-y · doi-reference
Enhancing translation: guidelines for standard pre-clinical experiments in mdx mice
10.1016/j.nmd.2011.04.012 · doi-reference
Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice
10.1038/nm.3628 · doi-reference
Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: a systematic review and meta-analysis
10.1002/mus.27682 · doi-reference
A new dystrophin-deficient rat model mirroring exon skipping in patients with DMD exon 45 deletions
10.1242/dmm.052578 · doi-reference
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
10.1073/pnas.240335297 · doi-reference
Myogenic reprogramming of bone marrow derived cells in a W41Dmdmdx deficient mouse model
10.1371/journal.pone.0027500 · doi-reference
Dystrophin/mini-dystrophin expression analysis by immunoaffinity liquid chromatography–tandem mass spectrometry after gene therapy for DMD
10.1038/s41434-025-00554-5 · doi-reference
A Duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to Exon 51 skipping
10.1371/journal.pone.0008647 · doi-reference
The first exon duplication mouse model of Duchenne muscular dystrophy: a tool for therapeutic development
10.1016/j.nmd.2015.08.005 · doi-reference
Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping
10.1038/mt.2012.181 · doi-reference
Seroprevalence of adeno-associated virus neutralizing antibodies in males with Duchenne muscular dystrophy
10.1089/hum.2022.081 · doi-reference
Quantitative muscle magnetic resonance outcomes in patients With Duchenne muscular dystrophy an exploratory analysis from the EMBARK randomized clinical trial
10.1001/jamaneurol.2025.0992 · doi-reference
Involvement of TRPC in the abnormal calcium influx observed in dystrophic (mdx) mouse skeletal muscle fibers
10.1083/jcb.200203091 · doi-reference
Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains
10.1016/j.nmd.2011.10.011 · doi-reference
Canine X-linked muscular dystrophy. An animal model of Duchenne muscular dystrophy: clinical studies
10.1016/0022-510x(88)90206-7 · doi-reference
Duchenne muscular dystrophy: recent insights in brain related comorbidities
10.1038/s41467-025-56644-w · doi-reference
Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase
10.1002/humu.20976 · doi-reference
Prevalence of muscular dystrophies: a systematic literature review
10.1159/000369343 · doi-reference
Pathological evaluation of rats carrying in-frame mutations in the dystrophin gene: a new model of Becker muscular dystrophy
10.1242/dmm.044701 · doi-reference
Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial
10.1212/wnl.0000000000006950 · doi-reference
Split intein-mediated protein trans-splicing to express large dystrophins
10.1038/s41586-024-07710-8 · doi-reference
Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical rat model identifies COMP as biomarker of fibrosis
10.1186/s40478-022-01355-2 · doi-reference
Eteplirsen: first global approval
10.1007/s40265-016-0657-1 · doi-reference
Duchenne Muscular dystrophy: a practice update
10.1007/s12098-017-2397-y · doi-reference
A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9
10.1242/dmm.032201 · doi-reference
Gene editing and modulation for Duchenne muscular dystrophy
10.1016/bs.pmbts.2021.01.029 · doi-reference
Analysis of NHEJ-based DNA repair after CRISPR-mediated DNA cleavage
10.3390/ijms22126397 · doi-reference
The molecular basis of muscular Dystrophy in the mdx mouse: a point mutation
10.1126/science.2662404 · doi-reference