Chemical synthesis and biological application of modified oligonucleotides
RNA plays a myriad of roles in the body including the coding, decoding, regulation, and
expression of genes. RNA oligonucleotides have garnered significant interest as …
expression of genes. RNA oligonucleotides have garnered significant interest as …
Splice-modulating antisense oligonucleotides as therapeutics for inherited metabolic diseases
Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …
Thiomorpholino oligonucleotides as a robust class of next generation platforms for alternate mRNA splicing
BT Le, S Paul, K Jastrzebska… - Proceedings of the …, 2022 - National Acad Sciences
Recent advances in drug development have seen numerous successful clinical translations
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …
Therapeutically significant microRNAs in primary and metastatic brain malignancies
Simple Summary The overall survival of brain cancer patients remains grim, with
conventional therapies such as chemotherapy and radiotherapy only providing marginal …
conventional therapies such as chemotherapy and radiotherapy only providing marginal …
Antisense oligonucleotide-mediated splice switching: potential therapeutic approach for cancer mitigation
Simple Summary Splicing is an important mechanism by which precursor mRNA is modified
into mature mRNA. This splicing plays a major role in the generation of different proteins …
into mature mRNA. This splicing plays a major role in the generation of different proteins …
Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro
Antisense oligonucleotide (AO)-mediated splice modulation has been established as a
therapeutic approach for tackling genetic diseases. Recently, Exondys51, a drug that aims to …
therapeutic approach for tackling genetic diseases. Recently, Exondys51, a drug that aims to …
Antisense oligonucleotides as potential therapeutics for type 2 diabetes
Type 2 diabetes (T2D) is a chronic metabolic disorder characterized by persistent
hyperglycemia resulting from inefficient signaling and insufficient production of insulin …
hyperglycemia resulting from inefficient signaling and insufficient production of insulin …
Evaluation of Chemically Modified Nucleic Acid Analogues for Splice Switching Application
In recent years, several splice switching antisense oligonucleotide (ASO)-based
therapeutics have gained significant interest, and several candidates received approval for …
therapeutics have gained significant interest, and several candidates received approval for …
Rational design of short locked nucleic acid-modified 2′-O-methyl antisense oligonucleotides for efficient exon-skipping in vitro
Locked nucleic acid is a prominent nucleic acid analog with unprecedented target binding
affinity to cDNA and RNA oligonucleotides and shows remarkable stability against nuclease …
affinity to cDNA and RNA oligonucleotides and shows remarkable stability against nuclease …
2′-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping
T Lee, H Awano, M Yagi, M Matsumoto, N Watanabe… - Genes, 2017 - mdpi.com
Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disease characterized by
dystrophin deficiency from mutations in the dystrophin gene. Antisense oligonucleotide (AO) …
dystrophin deficiency from mutations in the dystrophin gene. Antisense oligonucleotide (AO) …