Results 141 to 150 of about 29,161 (207)

Effect of chemical modification on the exon-skipping activity of heteroduplex oligonucleotides. [PDF]

Mol Ther Nucleic Acids
Shimo T, Hasegawa J, Yoshioka K, Nakatsuji Y, Aso K, Tachibana K, Nagata T, Yokota T, Obika S.   +8 more
europepmc   +1 more source

Preclinical development of genome editing to treat Duchenne muscular dystrophy by exon skipping. [PDF]

J Neuromuscul Dis
Padmaswari MH, Agrawal S, Nelson CE.
europepmc   +1 more source

DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function. [PDF]

Nat Commun
Shah MNA, Wilton-Clark H, Haque F, Powell B, Sutanto LE, Maradiya R, Zhabyeyev P, Roshmi RR, Anwar S, Aslesh T, Lim KRQ, Maruyama R, Bigot A, Young CS, Bittner S, Spencer MJ, Moulton HM, Oudit GY, Yokota T.   +18 more
europepmc   +1 more source

Valproic Acid Improves Antisense-Mediated Exon-Skipping Efficacy in <i>mdx</i> Mice. [PDF]

Int J Mol Sci
Phongsavanh M, Bizot F, Saoudi A, Gastaldi C, Le Coz O, Tensorer T, Brisebard E, Garcia L, Goyenvalle A.   +8 more
europepmc   +1 more source

Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy. [PDF]

J Muscle Res Cell Motil
Chwalenia K, Wood MJA, Roberts TC.
europepmc   +1 more source

Alternative spliceosomal protein Eftud2 mediated Kif3a exon skipping promotes SHH-subgroup medulloblastoma progression. [PDF]

Cell Death Differ
Li Y, Chen L, Xue S, Song Z, Liu H, Li H, Shen W, Zhang C, Wu H.   +8 more
europepmc   +1 more source

Protocol for using MYOD1-transduced human urine-derived cells as a predictive platform for exon skipping therapy in Duchenne muscular dystrophy. [PDF]

STAR Protoc
Kunitake K, Ishizuka T, Takeshita E, Komaki H, Aoki Y.   +4 more
europepmc   +1 more source

Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

Stenler S, Huang J, van Westering TL, Coenen-Stass AM, Krjutškov K, Andaloussi SE, Wood MJ, Roberts TC.   +7 more
europepmc   +1 more source

Novel FBN1 intron variant causes isolated ectopia lentis via in-frame exon skipping. [PDF]

J Hum Genet
Shimizu N, Mashimo Y, Yokouchi H, Nishio Y, Sawai S, Ichikawa T, Ogi T, Baba T, Onouchi Y.   +8 more
europepmc   +1 more source

Optimization of Exon-Skipping Riboswitches and Their Applications to Control Mammalian Cell Fate. [PDF]

ACS Synth Biol
Nomura Y, Kim N, Zhu B, Hamzah M, Zhang H, Yokobayashi Y.   +5 more
europepmc   +1 more source