Results 1 to 10 of about 21,845 (197)

MicroRNA-mRNA Networks in Skeletal Muscle of Tailored Pig Models for Dystrophinopathies. [PDF]

J Cachexia Sarcopenia Muscle
ABSTRACT Background Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are X‐linked dystrophinopathies caused by mutations in the dystrophin (DMD) gene. A common DMD‐causing mutation in humans is exon 52 deletion (DMDΔ52), which disrupts the reading frame and abolishes dystrophin expression.
Reschke S, Graf A, Hadlich F, Jaudas F, Fröhlich T, Stirm M, Klymiuk N, Krebs S, Wolf E, Ali A.   +9 more
europepmc   +2 more sources

Current and Emerging Therapeutic Strategies for the Treatment of Duchenne Muscular Dystrophy. [PDF]

Genes (Basel)
Lopez Perez MA, Weisleder NL.
europepmc   +1 more source

(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy: A Mechanistic and Transcriptomic Perspective. [PDF]

Degener Neurol Neuromuscul Dis
Remmel HL, Hammer SS, Blackburn SM, Quay SC.   +3 more
europepmc   +1 more source

Cytoplasmic region of beta-dystroglycan is essential for postsynaptic maturation and neuromuscular function in mice. [PDF]

Proc Natl Acad Sci U S A
Hord JM, Turk R, Kusano H, Rader EP, Burns S, Gastel Z, Prouty SJ, Yu L, Burden SJ, Campbell KP.   +9 more
europepmc   +1 more source

Role of Finerenone in Duchenne Muscular Dystrophy in a 45-Year-Old Man: Outcome and Brief Review. [PDF]

JACC Case Rep
Hillyer J, Jefferies JL, Silver MA.
europepmc   +1 more source

U7snRNA-mediated skipping of intron-derived pseudoexons restores full-length <i>DMD</i> expression in patient-derived cell lines. [PDF]

Mol Ther Adv
Beljan JC, Nicolau S, Lin H, Lay JW, Frair EC, Gushchina LV, Weiss RB, Flanigan KM.   +7 more
europepmc   +1 more source

Gene editing restores full-length dystrophin and affects iron homeostasis in hiPSC-derived cardiomyocytes from Becker Muscular Dystrophy patients. [PDF]

J Neuromuscul Dis
Przymuszała M, Kwiatkowska J, Meyer-Szary J, Śledzińska K, Wierzba J, Łoboda A, Stępniewski J, Florczyk-Soluch U, Dulak J.   +8 more
europepmc   +1 more source

Duchenne Muscular Dystrophy and Delandistrogene Moxeparvovec Gene Therapy in Children: A Systematic Review and Meta-Analysis. [PDF]

Neurol Genet
Antonello BB, Cargnelutti Fontoura F, Braga Albuquerque AL, Giovacchini G, Grespan Dill L, Valadão MC, Hadj Sadok S, Zattar Ribeiro PV.   +7 more
europepmc   +1 more source

Engineered exosomes improve myocardial cell membrane integrity and heart function in dystrophic cardiomyopathy. [PDF]

Clin Transl Med
Wu Q, Xue Y, Zhang K, Xu K, Xu T, Fu H, Zhang S, Zhang M, Sun R, Shan Chan SH, Zhou L, Cai X, Guo Y, Xu H.   +13 more
europepmc   +1 more source

Myocardial fat fraction in Becker muscular dystrophy and women carrying pathogenic <i>DMD</i> gene variants assessed by Dixon cardiac MRI. [PDF]

J Neuromuscul Dis
Lyu Z, Teitsdóttir B, Joensen H, Hjortrup MB, Slipsager A, Hansen AE, Vejlstrup N, Vissing J, Poulsen NS.   +8 more
europepmc   +1 more source