2024年1月5日 · Antisense oligonucleotides (ASOs) are incredibly versatile molecules that can be designed to specifically target and modify RNA transcripts to slow down or halt rare genetic …

Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and modulate protein expression through several different mechanisms. The ASO field is an emerging area …

ASOs are synthetic oligonucleotide or oligonucleotide analogs usually between 12 and 30 nucleotides in length that are designed to bind to RNA by Watson-Crick base pairing. ASOs …

2017年12月1日 · Two ASO-mediated splice-switching therapies for SMA and DMD have gained FDA approval within the past few years, putting neuromuscular diseases at the very forefront …

Antisense oligonucleotide (ASO)-based drugs are a new class of compounds. In this review, we will discuss ASO technology, including improved delivery systems, enhanced stability, …

2025年2月8日 · ASO therapy targets RNA molecules to modulate gene expression. ASOs are short, synthetic strands of nucleic acids designed to bind specifically to complementary RNA …

2025年4月2日 · BIIB080 is an investigational antisense oligonucleotide (ASO) therapy designed to target microtubule-associated protein tau (MAPT) mRNA to reduce the production of tau …

2025年4月1日 · Antisense Oligonucleotides (ASOs) are a modality of genetic therapy—they can bind to a patient’s native mRNA through Watson-Crick base pairing and subsequently alter …

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

5 天之前 · The FDA has granted a fast track designation to BIIB080 (Biogen Inc.), an investigational antisense oligonucleotide (ASO) therapy targeting tau, for the treatment of …