Results 211 to 220 of about 49,802 (297)

Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy. [PDF]

J Muscle Res Cell Motil
Chwalenia K, Wood MJA, Roberts TC.
europepmc   +1 more source

Ensemble-Learning and Feature Selection Techniques for Enhanced Antisense Oligonucleotide Efficacy Prediction in Exon Skipping. [PDF]

Pharmaceutics, 2023
Zhu A, Chiba S, Shimizu Y, Kunitake K, Okuno Y, Aoki Y, Yokota T, Yokota T.   +7 more
europepmc   +1 more source

Identification and Partial Characterization of a Variant of Human CXCR3 Generated by Posttranscriptional Exon Skipping [PDF]

, 2004
Jan E. Ehlert, Christina A. Addison, Marie D. Burdick, Steven L. Kunkel, Robert M. Strieter   +4 more
openalex   +1 more source

Protocol for using MYOD1-transduced human urine-derived cells as a predictive platform for exon skipping therapy in Duchenne muscular dystrophy. [PDF]

STAR Protoc
Kunitake K, Ishizuka T, Takeshita E, Komaki H, Aoki Y.   +4 more
europepmc   +1 more source

Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach. [PDF]

Mol Ther Nucleic Acids, 2023
Centa JL, Stratton MP, Pratt MA, Osterlund Oltmanns JR, Wallace DG, Miller SA, Weimer JM, Hastings ML.   +7 more
europepmc   +1 more source

Nuclear Factor TDP-43 Binds to the Polymorphic TG Repeats in CFTR Intron 8 and Causes Skipping of Exon 9: A Functional Link with Disease Penetrance [PDF]

, 2004
Emanuele Buratti, Antonia Brindisi, Franco Pagani, Francisco E. Baralle   +3 more
openalex   +1 more source

Novel FBN1 intron variant causes isolated ectopia lentis via in-frame exon skipping. [PDF]

J Hum Genet
Shimizu N, Mashimo Y, Yokouchi H, Nishio Y, Sawai S, Ichikawa T, Ogi T, Baba T, Onouchi Y.   +8 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

30 Years Since the Proposal of Exon Skipping Therapy for Duchenne Muscular Dystrophy and the Future of Pseudoexon Skipping. [PDF]

Int J Mol Sci
Matsuo M.
europepmc   +1 more source

Validation of the Idylla GeneFusion assay to detect fusions and MET exon-skipping in non-small cell lung cancers. [PDF]

Sci Rep, 2023
Gilson P, Pouget C, Belmonte R, Fadil S, Demange J, Rouyer M, Lacour J, Betz M, Dardare J, Witz A, Merlin JL, Harlé A.   +11 more
europepmc   +1 more source