Results 211 to 220 of about 30,223 (276)

Systematic deletion of symmetrical CFTR exons reveals new therapeutic targets for exon skipping antisense oligonucleotides. [PDF]

NAR Mol Med
Pena-Rasgado C, Rodriguez-Manriquez E, Dundr M, Bridges RJ, Hastings ML, Michaels WE.   +5 more
europepmc   +1 more source

A second hotspot for pathogenic exon-skipping variants in CDC45. [PDF]

Eur J Hum Genet
Schoch K, Ruegg MSG, Fellows BJ, Cao J, Uhrig S, Einsele-Scholz S, Biskup S, Hawarden SRA, Salpietro V, Capra V, Undiagnosed Diseases Network, Brown CM, Accogli A, Shashi V, Bicknell LS.   +14 more
europepmc   +1 more source

Rgr gene mutation and exon-skipping RGR mRNA in mice

, 2017
Zhaoxia Zhang, Henry Fong
openalex   +1 more source

Antisense oligonucleotide-mediated exon skipping strategies as the treatment for rare diseases

, 2020
Dunhui Li
openalex   +1 more source

Production of a heterozygous exon skipping model of common marmosets using gene-editing technology. [PDF]

Lab Anim (NY)
Sato K, Sasaguri H, Kumita W, Sakuma T, Morioka T, Nagata K, Inoue T, Kurotaki Y, Mihira N, Tagami M, Manabe RI, Ozaki K, Okazaki Y, Yamamoto T, Suematsu M, Saido TC, Sasaki E.   +16 more
europepmc   +1 more source

PP01.83 Treatment Sequencing in the VISION Study of Tepotinib in Patients with MET exon 14 (METex14) Skipping NSCLC [PDF]

Frank Griesinger, Marina Chiara Garassino, Enriqueta Felip, Hiroshi Sakai, Xiuning Le, Rémi Veillon, Egbert F. Smit, Jo Raskin, Michael Thomas, Myung‐Ju Ahn, Matthew Lee, Soetkin Vlassak, Rolf Bruns, Andreas Johne, Paul K. Paik   +14 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

Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse

, 2000
Christopher J. Mann
openalex   +1 more source