Új vizsgálatok, publikációk, preprintek és pályázatok ezen a célponton, hónapról hónapra.
Publikáció
Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials.
2026. okt. 29.· Journal of medical genetics· PUBMED
Publikáció
Teaching NeuroImage: Somatic DMD Gene Mosaicism Causing Hemiatrophy in an Adult Man With Duchenne Muscular Dystrophy.
2026. okt. 26.· Neurology· PUBMED
Publikáció
Statistical correlation between strength and mini-dystrophin expression in skeletal muscle after an rAAV9-mini-dystrophin gene therapy candidate in DMDmdx rats.
2026. okt. 17.· Gene therapy· PUBMED
Publikáció
Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.
2026. okt. 1.· Experimental & molecular medicine· PUBMED
Publikáció
From molecular correction to functional rescue: delivery, tissue state, and immunobiology as the principal constraints on dystrophin-restoring therapy in Duchenne muscular dystrophy.
2026. szept. 22.· Frontiers in pharmacology· PUBMED
Publikáció
Mutational Spectrum of DMD Gene Variants and Cascade Screening Outcomes in the Central-Eastern Black Sea Region of Türkiye: A Single-Centre Retrospective Study.
2026. szept. 22.· Molecular biology reports· PUBMED
Publikáció
Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy.
2026. szept. 10.· Molecular therapy : the journal of the American Society of Gene Therapy· PUBMED
Publikáció
Proteomics of the skeletal muscle dystrophin complex.
2026. szept. 8.· Advances in clinical chemistry· PUBMED
Vizsgálat
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
2026. szept. 1.· PHASE3 · RECRUITING· CTGOV
Publikáció
Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.
2026. aug. 28.· Disease models & mechanisms· PUBMED
Publikáció
Exon-skipping therapies for DMD in Kazakhstan: Progress and challenges.
2026. aug. 27.· Journal of neuromuscular diseases· PUBMED
Publikáció
Characterization of Dystrophin-Related Syndromes: Carriers, DMD, and BMD.
2026. aug. 26.· Muscles (Basel, Switzerland)· PUBMED
Publikáció
Beyond dystrophin: cell therapy for Duchenne muscular dystrophy.
2026. aug. 22.· Lancet (London, England)· PUBMED
Publikáció
Evaluation of antibody reactivity to dystrophin products: does Dp40 really exist?
2026. aug. 4.· Neuromuscular disorders : NMD· PUBMED
Publikáció
Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to DMD exon 16 skipping.
2026. aug. 3.· Molecular therapy. Nucleic acids· PUBMED
Publikáció
Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons.
2026. júl. 31.· Genome medicine· PUBMED
Vizsgálat
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping
2026. júl. 28.· PHASE3 · RECRUITING· CTIS
Publikáció
A high-fat, high-sucrose diet exacerbates muscle and metabolic pathology and undermines glucocorticoid efficacy in dystrophin-deficient mice.
2026. júl. 1.· The Journal of physiology· PUBMED
Publikáció
Dystrophin-Deficient Muscular Dystrophy in a Jack Russell Terrier With a Large Deletion in the Canine DMD Gene.
2026. júl. 1.· Veterinary medicine and science· PUBMED
Publikáció
Novel DMD Frameshift Variant (p.Leu2017Profs*5) in Spectrin-Like Repeat 16 Expands the Mutational Spectrum of DMD.
2026. júl. 1.· Molecular genetics & genomic medicine· PUBMED
Publikáció
Calcium handling properties and arrhythmia vulnerability of cardiomyocytes from dystrophin-deficient mdx mice.
2026. jún. 22.· Cell calcium· PUBMED
Publikáció
Muscle-targeted extracellular vesicles for full-length dystrophin mRNA therapy in Duchenne muscular dystrophy.
2026. jún. 11.· Nature biomedical engineering· PUBMED
Publikáció
Exploring Dystrophin Expression and Mutations in the DMD and Dystrophin-Glycoprotein Complex Genes as Prognostic Factors in Leiomyosarcomas.
2026. jún. 11.· International journal of molecular sciences· PUBMED
Publikáció
Gene editing restores full-length dystrophin and affects iron homeostasis in hiPSC-derived cardiomyocytes from Becker Muscular Dystrophy patients.
2026. jún. 5.· Journal of neuromuscular diseases· PUBMED
Publikáció
Microbiota dysbiosis influences immune system and muscle pathophysiology of dystrophin deficient mice.
2026. jún. 3.· EMBO molecular medicine· PUBMED
Preprint
Stress unmasks impaired endocrine coordination of glucose metabolism in dystrophin deficiency.
2026. máj. 27.· bioRxiv : the preprint server for biology· PUBMED
Publikáció
A female manifesting carrier of DMD with exon 45 deletion: a case report.
2026. máj. 25.· Translational pediatrics· PUBMED
Publikáció
Reply to the letter of editor addressed by Thulamy Raman D. Et al., about our article entitled "Radiosensitivity and delayed radiation-induced Nucleo-shuttling of the ATM protein in fibroblasts from Duchenne muscular dystrophy expressing residual dystrophin".
2026. máj. 23.· Journal of the neurological sciences· PUBMED
Publikáció
Comment on "Radiosensitivity and delayed radiation-induced nucleo-shuttling of the ATM protein in fibroblasts from Duchenne muscular dystrophy expressing residual dystrophin".
2026. máj. 22.· Journal of the neurological sciences· PUBMED
Publikáció
Quantification of a serum titin fragment reflects dystrophin restoration in mdx mice and disease severity in patients with dystrophinopathies.
2026. máj. 20.· Journal of neuromuscular diseases· PUBMED