Results 111 to 120 of about 40,676 (197)

RNA Therapeutics for Duchenne Muscular Dystrophy: Exon Skipping, RNA Editing, and Translational Insights from Genome-Edited Microminipig Models. [PDF]

Int J Mol Sci
Chassin A, Ono H, Ashida Y, Imamura M, Aoki Y.   +4 more
europepmc   +1 more source

Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Blitek M, Gastaldi C, Doisy M, Le Coz O, David M, Phongsavanh X, Ben Aicha S, Garcia L, Rotini A, Pagès G, Goyenvalle A.   +10 more
europepmc   +1 more source

Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy [PDF]

A Asai, A Crisp, A De Troyer, A Malerba, A Malerba, A Malerba, BJ Petrof, C Betts, C Kirchmann, CF Spurney, DJ Stuckey, DM Connuck, G Dickson, G Nigro, H Yin, H Yin, J Alter, J Kuroda, J Morrison, JG Quinlan, JM Costas, JO Benditt, JW Carnwath, K Ito, K Ohlendieck, L Loufrani, L Loufrani, L Loufrani, M Ishizaki, M Ishizaki, M Mosqueira, M Palladino, MG Dunckley, N Jearawiriyapaisarn, P Melacini, QL Lu, S Fletcher, SD Wilton   +37 more
core   +1 more source

A novel non-sense variant in <i>GSDME</i> causing exon skipping associated with DFNA5 in a large Chinese family. [PDF]

Front Neurol
Yang B, Zhu M, Long X, Wan S, Lu Y, Yuan H, Hua Q.   +6 more
europepmc   +1 more source

Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy. [PDF]

Brain
Moschovaki-Filippidou F, de Carvalho Neves J, Diedhiou N, Jad Y, Böhm J, Wood MJA, Varela MA, Laporte J.   +7 more
europepmc   +1 more source

A new dystrophin-deficient rat model mirroring exon skipping in patients with DMD exon 45 deletions. [PDF]

Dis Model Mech
Wang T, Daoud C, Dubois A, Corre G, Bellec J, Bovolenta M, Philidet L, Dorval A, Bourg N, Roudaut C, Albini S, Warthi G, Jaber A, Richard I.   +13 more
europepmc   +1 more source

From Mutation to Manifestation: Evaluation of a <i>PKLR</i> Gene Truncation Caused by Exon Skipping in a Schnauzer Terrier. [PDF]

Animals (Basel)
Ma TY, Kuo CJ, Liu PC.
europepmc   +1 more source

Exon Skipping Therapy Restores Ciliary Function in USH2A-Related Retinal Degeneration. [PDF]

Invest Ophthalmol Vis Sci
Li W, Li Y, Zhou Y, Liu Y, Liu H, Wei X, Sun Z, Han X, Zou X, Li H, Sui R.   +10 more
europepmc   +1 more source

Exon-skipping due to bi-allelic splice site mutations in the neurodevelopmental disease gene LNPK. [PDF]

HGG Adv
Doss RM, Wirth SA, Pitsch JW, Dias CM, Gropman AL, Breuss MW.   +5 more
europepmc   +1 more source

Target choice and exon skipping regulate CRISPR-directed gene editing of NRF2 in head/neck and esophageal cancer cells. [PDF]

Mol Ther Oncol
Rivera-Torres N, Skelly LE, Rogowskyj JA, Aguilar G, Banas K, Bialk P, Kmiec EB.   +6 more
europepmc   +1 more source