Results 91 to 100 of about 21,009 (196)

Dystrophin [PDF]

, 2020
openaire   +1 more source

FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook. [PDF]

RNA Biol
Moriyama H, Moriyama S, Yokota T.
europepmc   +1 more source

Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translation. [PDF]

Cells
Siddika A, Rousseau J, Veillette F, Bouchard C, Lu Y, Tremblay JP.   +5 more
europepmc   +1 more source

The atomic structure of human dystrophin spectrin-like repeat 24. [PDF]

Acta Crystallogr F Struct Biol Commun
Streeter O, Shi K, Bui H, Aihara H, Ervasti JM, Evans R, Muretta JM.   +6 more
europepmc   +1 more source

Genetic cardiomyopathy unmasked by pregnancy: X-linked dystrophinopathy presenting as peripartum cardiomyopathy-a case report. [PDF]

Eur Heart J Case Rep
Bauer J, Knebel F, Haverkamp W, Klingel K, Aleksandrov A.   +4 more
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

Duchenne muscular dystrophy coexisting with Down syndrome or Turner syndrome: Two case reports. [PDF]

World J Clin Pediatr
Pongsakornkullachart P, Chanvanichtrakool M, Dhachpramuk D, Pho-Iam T, Yongwattana N, Kulsirichawaroj P, Tanboon J, Sanmaneechai O.   +7 more
europepmc   +1 more source

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

Genes (Basel)
Lopez Perez MA, Weisleder NL.
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

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