Novel splice‐switching oligonucleotide promotes BRCA1 aberrant splicing and susceptibility to PARP inhibitor action
Sign Up to like & getrecommendations! Published in 2017 at "International Journal of Cancer"
DOI: 10.1002/ijc.30574
Abstract: Tumors carrying hereditary mutations in BRCA1, which attenuate the BRCA1 DNA damage repair pathway, are more susceptible to dual treatment with PARP inhibitors and DNA damaging therapeutics. Conversely, breast cancer tumors with nonmutated functional BRCA1… read more here.
Keywords: susceptibility parp; brca1; parp inhibitor; splice switching ... See more keywords
Regulation of Tak1 alternative splicing by splice-switching oligonucleotides.
Sign Up to like & getrecommendations! Published in 2018 at "Biochemical and biophysical research communications"
DOI: 10.1016/j.bbrc.2018.02.160
Abstract: Alternative splicing (AS) generates multiple isoforms from a single precursor mRNA, and these isoforms usually exhibit different tissue distributions and functions. Aberrant protein isoforms can lead to abnormalities in protein function and may even result… read more here.
Keywords: tak1; switching oligonucleotides; splice switching; alternative splicing ... See more keywords
Inhibition of nuclease activity by a splice-switching oligonucleotide targeting deoxyribonuclease 1 mRNA prevents apoptosis progression and prolong viability of normal human CD4+ T-lymphocytes.
Sign Up to like & getrecommendations! Published in 2020 at "Biochimie"
DOI: 10.1016/j.biochi.2020.04.009
Abstract: The nuclease activity of deoxyribonuclease 1 (DNase I) is regulated by alternative splicing (AS) of its mRNA. The aim of this study was to define the ability of a splice-switching oligonucleotide (SSO) that base-paired with… read more here.
Keywords: splice switching; nuclease activity; activity; mrna ... See more keywords
Splice-switching as cancer therapy.
Sign Up to like & getrecommendations! Published in 2021 at "Current opinion in pharmacology"
DOI: 10.1016/j.coph.2021.05.008
Abstract: In light of recent advances in RNA splicing modulation as therapy for specific genetic diseases, there is great optimism that this approach can be applied to treatment of cancer as well. Dysregulation of alternative RNA… read more here.
Keywords: therapy; switching cancer; splice switching; cancer therapy ... See more keywords
Thiomorpholino oligonucleotides as a robust class of next generation platforms for alternate mRNA splicing
Sign Up to like & getrecommendations! Published in 2022 at "Proceedings of the National Academy of Sciences of the United States of America"
DOI: 10.1073/pnas.2207956119
Abstract: Significance Splice-switching antisense oligonucleotides (ASOs) represent a unique class of drug molecules with the US Food and Drug Administration approval of Exondys 51, Vyondys 53, Amondys 45, and Viltepso for the treatment of Duchenne muscular… read more here.
Keywords: splice switching; oligonucleotides robust; class; thiomorpholino oligonucleotides ... See more keywords
AON-induced splice-switching and DMPK pre-mRNA degradation as potential therapeutic approaches for Myotonic Dystrophy type 1
Sign Up to like & getrecommendations! Published in 2020 at "Nucleic Acids Research"
DOI: 10.1093/nar/gkaa007
Abstract: Abstract Expansion of an unstable CTG repeat in the 3′UTR of the DMPK gene causes Myotonic Dystrophy type 1 (DM1). CUG-expanded DMPK transcripts (CUGexp) sequester Muscleblind-like (MBNL) alternative splicing regulators in ribonuclear inclusions (foci), leading… read more here.
Keywords: dmpk; dystrophy type; chemistry; myotonic dystrophy ... See more keywords
Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations
Sign Up to like & getrecommendations! Published in 2023 at "RNA"
DOI: 10.1261/rna.079540.122
Abstract: Splice-modulating antisense oligonucleotides (ASOs) offer treatment options for rare neurological diseases, including those with very rare mutations, where patient-specific, individualized ASOs have to be developed. Inspired by the development of milasen, the 1 Mutation 1… read more here.
Keywords: splice switching; splice; development tailored; development ... See more keywords
Delivery is key: lessons learnt from developing splice‐switching antisense therapies
Sign Up to like & getrecommendations! Published in 2017 at "EMBO Molecular Medicine"
DOI: 10.15252/emmm.201607199
Abstract: The use of splice‐switching antisense therapy is highly promising, with a wealth of pre‐clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety of disorders has yet to be realized. The… read more here.
Keywords: key lessons; delivery; splice switching; delivery key ... See more keywords
Fine Tuning of Phosphorothioate Inclusion in 2′-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation
Sign Up to like & getrecommendations! Published in 2021 at "Frontiers in Physiology"
DOI: 10.3389/fphys.2021.689179
Abstract: Splice-switching antisense oligonucleotide- (SSO-) mediated correction of framedisrupting mutation-containing premessenger RNA (mRNA) transcripts using exon skipping is a highly promising treatment method for muscular diseases such as Duchenne muscular dystrophy (DMD). Phosphorothioate (PS) chemistry, a… read more here.
Keywords: splice; splice switching; cell; inclusion methyl ... See more keywords
Splice-Switching Therapy for Spinal Muscular Atrophy
Sign Up to like & getrecommendations! Published in 2017 at "Genes"
DOI: 10.3390/genes8060161
Abstract: Spinal muscular atrophy (SMA) is a genetic disorder with severity ranging from premature death in infants to restricted motor function in adult life. Despite the genetic cause of this disease being known for over twenty… read more here.
Keywords: spinal muscular; switching therapy; splice switching; muscular atrophy ... See more keywords