Cargando…
Construction of a tri-chromatic reporter cell line for the rapid and simple screening of splice-switching oligonucleotides targeting DMD exon 51 using high content screening
Splice-switching oligonucleotides (SSOs) that can modulate RNA splicing are used for the treatment of many genetic disorders. To enhance the efficacy of modulating splicing, it is important to optimize SSOs with regard to target sites, GC content, melting temperature (T(m) value), chemistries, and l...
Autores principales: | Shimo, Takenori, Tachibana, Keisuke, Obika, Satoshi |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2018
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5955590/ https://www.ncbi.nlm.nih.gov/pubmed/29768479 http://dx.doi.org/10.1371/journal.pone.0197373 |
Ejemplares similares
-
Design and In Vitro Evaluation of Splice-Switching Oligonucleotides Bearing Locked Nucleic Acids, Amido-Bridged Nucleic Acids, and Guanidine-Bridged Nucleic Acids
por: Shimo, Takenori, et al.
Publicado: (2021) -
Design and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro
por: Shimo, Takenori, et al.
Publicado: (2014) -
Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD
por: Akpulat, Ugur, et al.
Publicado: (2018) -
Abnormal splicing switch of DMD's penultimate exon compromises muscle fibre maintenance in myotonic dystrophy
por: Rau, Frédérique, et al.
Publicado: (2015) -
Systemic deletion of DMD exon 51 rescues clinically severe Duchenne muscular dystrophy in a pig model lacking DMD exon 52
por: Stirm, Michael, et al.
Publicado: (2023)