Results 261 to 270 of about 240,839 (324)

Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping. [PDF]

Mol Ther Methods Clin Dev, 2023
Wang P, Li H, Zhu M, Han RY, Guo S, Han R.   +5 more
europepmc   +1 more source

638. Therapeutic Antisense-Induced Exon Skipping for Duchenne Muscular Dystrophy [PDF]

, 2004
Annemieke Aartsma‐Rus, Anneke A. M. Janson, Wendy E. Kaman, Mattie Bremmer-Bout, C.L. de Winter, Habte F. Teshale, Gert‐Jan B. van Ommen, Johan T. den Dunnen, J.C.T. van Deutekom   +8 more
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

Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model. [PDF]

Cells, 2023
Bizot F, Fayssoil A, Gastaldi C, Irawan T, Phongsavanh X, Mansart A, Tensorer T, Brisebard E, Garcia L, Juliano RL, Goyenvalle A.   +10 more
europepmc   +1 more source

Erratum: A novel splice site mutation (3157+1G>T) in the dystrophin gene causing total exon skipping and DMD phenotype

, 2001
Manuela Sironi, Stefania Corti, Federica Locatelli, Rachele Cagliani, Giacomo P. Comi   +4 more
openalex   +1 more source

The Alu insertion in the CLCN5 gene of a patient with Dent’s disease leads to exon 11 skipping [PDF]

, 2005
Félix Claveríe‐Martín, Carlos Flores, Montserrat Antón Gamero, Hilaria González-Acosta, Víctor Manuel García Nieto   +4 more
openalex   +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

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. [PDF]

Nucleic Acid Ther, 2023
Aartsma-Rus A, Garanto A, van Roon-Mom W, McConnell EM, Suslovitch V, Yan WX, Watts JK, Yu TW.   +7 more
europepmc   +1 more source

A Duplication in the Canine β-Galactosidase Gene GLB1 Causes Exon Skipping and GM1-Gangliosidosis in Alaskan Huskies [PDF]

, 2005
Robert Kreutzer, Tosso Leeb, G. Müller, Andreas Moritz, Wolfgang Baumgärtner   +4 more
openalex   +1 more source

Reversing T Cell Exhaustion by Converting Membrane PD-1 to Its Soluble form in Jurkat Cells; Applying The CRISPR/Cas9 Exon Skipping Strategy. [PDF]

Cell J, 2023
Yousefi-Najafabadi Z, Mehmandoostli Z, Asgari Y, Kaboli S, Falak R, Kardar GA.   +5 more
europepmc   +1 more source