Results 151 to 160 of about 40,676 (197)

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

Twin Prime Editing Mediated Exon Skipping/Reinsertion for Restored Collagen VII Expression in Recessive Dystrophic Epidermolysis Bullosa. [PDF]

J Invest Dermatol
Steinbeck BJ, Gao XD, McElroy AN, Pandey S, Doman JL, Riddle MJ, Xia L, Chen W, Eide CR, Lengert AH, Han SW, Blazar BR, Wandall HH, Dabelsteen S, Liu DR, Tolar J, Osborn MJ.   +16 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

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

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Exon-skipping therapy for Duchenne muscular dystrophy

The Lancet, 2009
Duchenne muscular dystrophy (DMD) is a lethal muscle disorder caused by mutations in the DMD gene for which no mutation‐targeted therapy has been available thus far. However, exon‐skipping mediated by antisense oligonucleotides (AOs), which are short single‐strand DNAs, has considerable potential for DMD therapy, and clinical trials in DMD patients are
Akinori, Nakamura, Shin'ichi, Takeda
openaire   +3 more sources

DNA Diagnostics and Exon Skipping

2012
The predominate form of DNA diagnostics remains nucleic acid sequencing in the research and clinical setting. While DNA sequencing allows a mutation to be correctly identified, only RNA sequencing can confirm the effect of that mutation on the resulting mRNA transcript.
Umasuthan, Srirangalingam, Shern L, Chew
openaire   +2 more sources

Exon Skipping Mutations in Neurofibromatosis

2012
Defects at the level of pre-mRNA splicing represent a common source of disease mutations in almost all known diseases with a genetic aetiology. In general, it is commonly accepted that 15% of all pathogenic mutations are caused by splicing defects. However, this is probably a conservative estimate since clinical practice has only recently begun to ...
Buratti, Emanuele, Baralle, Diana
openaire   +3 more sources