Ontology highlight
ABSTRACT:
INSTRUMENT(S): Illumina HiSeq 1500
ORGANISM(S): Mus musculus
SUBMITTER: Paul Wilson
PROVIDER: E-MTAB-5180 | biostudies-arrayexpress |
REPOSITORIES: biostudies-arrayexpress
Kamola Piotr J PJ Maratou Klio K Wilson Paul A PA Rush Kay K Mullaney Tanya T McKevitt Tom T Evans Paula P Ridings Jim J Chowdhury Probash P Roulois Aude A Fairchild Ann A McCawley Sean S Cartwright Karen K Gooderham Nigel J NJ Gant Timothy W TW Moores Kitty K Hughes Stephen A SA Edbrooke Mark R MR Clark Kenneth K Parry Joel D JD
Molecular therapy. Nucleic acids 20170708
Antisense oligonucleotide (ASO) gapmers downregulate gene expression by inducing enzyme-dependent degradation of targeted RNA and represent a promising therapeutic platform for addressing previously undruggable genes. Unfortunately, their therapeutic application, particularly that of the more potent chemistries (e.g., locked-nucleic-acid-containing gapmers), has been hampered by their frequent hepatoxicity, which could be driven by hybridization-mediated interactions. An early de-risking of this ...[more]