Antisense oligonucleotides and their applications in rare neurological diseases
Rare diseases affect almost 500 million people globally, predominantly impacting children
and often leading to significantly impaired quality of life and high treatment costs. While …
and often leading to significantly impaired quality of life and high treatment costs. While …
Precision medicine through antisense oligonucleotide-mediated exon skipping
Clinical implementation of two recently approved antisense RNA therapeutics–Exondys51®
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
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 …
Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era
Parkinson's disease (PD) is one of the most common neurodegenerative disorders that
manifest various motor and nonmotor symptoms. Although currently available therapies can …
manifest various motor and nonmotor symptoms. Although currently available therapies can …
Thrombin binding aptamer G-quadruplex stabilized by pyrene-modified nucleotides
M Kovačič, P Podbevšek… - Nucleic Acids …, 2020 - academic.oup.com
Guanine-rich regions of the human genome can adopt non-canonical secondary structures.
Their role in regulating gene expression has turned them into promising targets for …
Their role in regulating gene expression has turned them into promising targets for …
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 …
Recent advances in nucleic acid targeting probes and supramolecular constructs based on pyrene-modified oligonucleotides
In this review, we summarize the recent advances in the use of pyrene-modified
oligonucleotides as a platform for functional nucleic acid-based constructs. Pyrene is of …
oligonucleotides as a platform for functional nucleic acid-based constructs. Pyrene is of …
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 …
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 …
Antisense oligonucleotide modified with serinol nucleic acid (SNA) induces exon skipping in mdx myotubes
Serinol nucleic acid (SNA) is a novel nucleic acid analogue that can form highly stable
heteroduplexes with complementary DNA and RNA sequences. Structurally, SNA is a close …
heteroduplexes with complementary DNA and RNA sequences. Structurally, SNA is a close …