Results 1 to 10 of about 32,246 (220)

Bridging Duchenne muscular dystrophy therapy from rhesus monkeys to patients. [PDF]

Clin Transl Med
Guo W, Ji W, Chen Y.
europepmc   +1 more source

Givinostat enhances antisense oligonucleotide efficacy in the <i>mdx52</i> model of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Phongsavanh X, Gastaldi C, Mottais A, Doisy M, Le Coz O, de Haan S, Spitali P, Garcia L, Goyenvalle A.   +8 more
europepmc   +1 more source

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. [PDF]

Genome Med
Rovina D, Milasi S, Di Giuseppe G, Pioner JM, Langione M, Giammarino L, Ferrantini C, Chiesa M, Canonico F, D'Amario D, Mercuri EM, Pompilio G, Sommariva E, Gowran A, Maione AS.   +14 more
europepmc   +1 more source

Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to <i>DMD</i> exon 16 skipping. [PDF]

Mol Ther Nucleic Acids
Geoffroy-Guiraud M, Beley C, Campuzano S, Nguyen TH, Schmidt LM, Blaszykowski C, Krueger M, Garcia L, Guiraud S.   +8 more
europepmc   +1 more source

Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Stenler S, Huang J, van Westering TLE, Coenen-Stass AML, Jad Y, Krjutškov K, El Andaloussi S, Wood MJA, Roberts TC.   +8 more
europepmc   +1 more source

Congenital Clubfoot as an Early Manifestation of Duchenne Muscular Dystrophy? [PDF]

Muscles
Kenis V, Shpulev P, Melnik E, Sosnina I, Snegova E.   +4 more
europepmc   +1 more source

Clinical, pathological, and genetic characteristics of 23 DMD patients in northern China. [PDF]

Orphanet J Rare Dis
Bu Y, Han J, Deng J, Fang P, An D, Ji G, Ma S, Song X.   +7 more
europepmc   +1 more source

Exploring Dystrophin Expression and Mutations in the <i>DMD</i> and Dystrophin-Glycoprotein Complex Genes as Prognostic Factors in Leiomyosarcomas. [PDF]

Int J Mol Sci
Salazar J, Cerdà P, Artigas A, Orellana R, González A, Terés R, Arranz MJ, Bagué S, Fumagalli C, González JA, Peiró A, González-Quereda L, Rodríguez MJ, Gallardo E, Aguado M, Sebio A.   +15 more
europepmc   +1 more source

Microdystrophins partially rescue deficits of Duchenne muscular dystrophy iPSC-cardiomyocytes. [PDF]

Mol Ther Adv
Keegan AR, Gonzalez AFGS, Zhang R, Doan H, Torres-Bigio SI, Lau F, Ly KN, Lai EC, Mamuyac LC, Bhimaraju S, Mishra A, Polen MK, Ortiz DS, Lam JH, Eguchi A.   +14 more
europepmc   +1 more source

Dystrophin-Deficient Muscular Dystrophy in a Jack Russell Terrier With a Large Deletion in the Canine DMD Gene. [PDF]

Vet Med Sci
Royaux E, Shelton GD, Jagannathan V, Leeb T, Drögemüller M.   +4 more
europepmc   +1 more source