Matches in SemOpenAlex for { <https://semopenalex.org/work/W2789323621> ?p ?o ?g. }
- W2789323621 abstract "Antisense oligonucleotides are an emerging therapeutic option to treat diseases with known genetic origin. In the age of personalised medicines, antisense oligonucleotides can sometimes be designed to target and bypass or overcome a patient’s genetic mutation, in particular those lesions that compromise normal pre-mRNA processing. Antisense oligonucleotides can alter gene expression through a variety of mechanisms as determined by the chemistry and antisense oligomer design. Through targeting the pre-mRNA, antisense oligonucleotides can alter splicing and induce a specific spliceoform or disrupt the reading frame, target an RNA transcript for degradation through RNaseH activation, block ribosome initiation of protein translation or disrupt miRNA function. The recent accelerated approval of eteplirsen (renamed Exondys 51™) by the Food and Drug Administration, for the treatment of Duchenne muscular dystrophy, and nusinersen, for the treatment of spinal muscular atrophy, herald a new and exciting era in splice-switching antisense oligonucleotide applications to treat inherited diseases. This review considers the potential of antisense oligonucleotides to treat inherited lung diseases of childhood with a focus on cystic fibrosis and disorders of surfactant protein metabolism." @default.
- W2789323621 created "2018-03-29" @default.
- W2789323621 creator A5022309517 @default.
- W2789323621 creator A5022772543 @default.
- W2789323621 creator A5035023951 @default.
- W2789323621 creator A5038285432 @default.
- W2789323621 creator A5061703216 @default.
- W2789323621 creator A5070934428 @default.
- W2789323621 creator A5080459494 @default.
- W2789323621 date "2018-02-06" @default.
- W2789323621 modified "2023-09-25" @default.
- W2789323621 title "The potential of antisense oligonucleotide therapies for inherited childhood lung diseases" @default.
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