Results 241 to 250 of about 49,802 (297)

Rescue of Dystrophic Muscle Through U7 snRNA-Mediated Exon Skipping

, 2004
Aurélie Goyenvalle, Adeline Vulin, Françoise Fougerousse, France Leturcq, Jean‐Claude Kaplan, Luis Garcı́a, Olivier Danos   +6 more
openalex   +1 more source

Targeted Exon Skipping in Transgenic hDMD Mice: A Model for Direct Preclinical Screening of Human-Specific Antisense Oligonucleotides

, 2004
Mattie Bremmer-Bout, Annemieke Aartsma‐Rus, Emile J. de Meijer, Wendy E. Kaman, Anneke A. M. Janson, Rolf H. A. M. Vossen, Gert‐Jan B. van Ommen, Johan T. den Dunnen, J.C.T. van Deutekom   +8 more
openalex   +1 more source

Is Exon Skipping a Viable Therapeutic Approach for Vascular Ehlers-Danlos Syndrome with Mutations in COL3A1 Exon 10 or 15? [PDF]

Int J Mol Sci
Utama S, Cale JM, Mitrpant C, Fletcher S, Wilton SD, Aung-Htut MT.   +5 more
europepmc   +1 more source

Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping. [PDF]

Mol Ther Nucleic Acids
McCormack NM, Calabrese KA, Sun CM, Tully CB, Heier CR, Fiorillo AA.   +5 more
europepmc   +1 more source

Adenine base editing-mediated exon skipping restores dystrophin in humanized Duchenne mouse model. [PDF]

Nat Commun
Lin J, Jin M, Yang D, Li Z, Zhang Y, Xiao Q, Wang Y, Yu Y, Zhang X, Shao Z, Shi L, Zhang S, Chen WJ, Wang N, Wu S, Yang H, Xu C, Li G.   +17 more
europepmc   +1 more source

A Comprehensive Analysis of CSN1S2 I and II Transcripts Reveals Significant Genetic Diversity and Allele-Specific Exon Skipping in Ragusana and Amiatina Donkeys. [PDF]

Animals (Basel)
Cosenza G, Pauciullo A.
europepmc   +1 more source

Highly efficient CRISPR/Cas9-mediated exon skipping for recessive dystrophic epidermolysis bullosa. [PDF]

Bioeng Transl Med
du Rand A, Hunt J, Samson C, Loef E, Malhi C, Meidinger S, Chen CJ, Nutsford A, Taylor J, Dunbar R, Purvis D, Feisst V, Sheppard H.   +12 more
europepmc   +1 more source

Author Correction: Cell-mediated exon skipping normalizes dystrophin expression and muscle function in a new mouse model of Duchenne Muscular Dystrophy. [PDF]

EMBO Mol Med
Galli F, Bragg L, Rossi M, Proietti D, Perani L, Bacigaluppi M, Tonlorenzi R, Sibanda T, Caffarini M, Talapatra A, Santoleri S, Meregalli M, Bano-Otalora B, Bigot A, Bozzoni I, Bonini C, Mouly V, Torrente Y, Cossu G.   +18 more
europepmc   +1 more source

Cellular senescence induced by down-regulation of PTBP1 correlates with exon skipping of mitochondrial-related gene NDUFV3. [PDF]

Life Med
Yang Y, Wen H, Li Y, Zeng X, Wei G, Gu Z, Ni T.   +6 more
europepmc   +1 more source

Cell-mediated exon skipping normalizes dystrophin expression and muscle function in a new mouse model of Duchenne Muscular Dystrophy. [PDF]

EMBO Mol Med
Galli F, Bragg L, Rossi M, Proietti D, Perani L, Bacigaluppi M, Tonlorenzi R, Sibanda T, Caffarini M, Talapatra A, Santoleri S, Meregalli M, Bano-Otalora B, Bigot A, Bozzoni I, Bonini C, Mouly V, Torrente Y, Cossu G.   +18 more
europepmc   +1 more source