Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA ...

Antisense therapy is a way to treat ALS and other neurologic conditions using short DNA-like molecules called antisense oligonucleotides (ASOs).

Antisense therapy is an approach to fighting diseases using short DNA-like molecules called antisense oligonucleotides. Recently, antisense therapy has emerged as an exciting and promising strategy for the treatment of various neurodegenerative and neuromuscular disorders.

Antisense therapy involves administering antisense oligonucleotides (ASOs) to targets and inhibiting the translation of disease-associated messenger RNA (mRNA) . ASOs are short, single-stranded, synthetic polymer nucleotides that regulate gene expression by gene silencing or modulating splicing [ 2 ].

2021年3月24日 · Nine single-stranded antisense oligonucleotide (ASO) drugs representing four chemical classes, two mechanisms of action and four routes of administration have been approved for commercial use,...

Antisense oligonucleotides (ASOs) are short oligonucleotides that modify gene expression and mRNA splicing in the nervous system. The FDA has approved ASOs for treatment of ten genetic disorders, with many applications currently in the pipeline.

Antisense therapy delivers antisense molecules that target to mRNA to the target cells with, which they can hybridize and specifically inhibit the expression of target genes. Antisense oligonucleotides have been widely used in investigating gene functions and regulation.

2022年2月25日 · Antisense technology can be extremely useful to degrade transcripts and cause gene silencing (knockdown). From the therapeutic perspective, this might be a potential tool to treat autosomal dominant diseases caused by dominant-negative mutations.

2024年1月11日 · Downregulating superoxide dismutase 1 (SOD1) by regular injections of antisense oligos (ASOs) interacting with SOD1 mRNA is a new FDA-approved therapy for patients with amyotrophic lateral sclerosis (ALS) with mutations in SOD1.

2019年11月20日 · Optimization of enhanced delivery, specificity, affinity, and nuclease resistance with reduced toxicity is underway in different disease context. This chapter gives a complete overview of antisense therapy and highlights its potential. Here, we focused on the advances of the antisense technology, pharmacology, therapeutics, and drug discovery.