Antisense oligonucleotides (ASOs) have emerged as a new class of drugs to treat a wide range of diseases, including neurological indications. Spinraza, an ASO that modulates splicing of SMN2 RNA, has shown profound disease modifying effects in Spinal Muscular Atrophy (SMA) patients, energizing efforts to develop ASOs for other neurological
Antisense RNA ( asRNA ), also referred to as antisense transcript, [1] natural antisense transcript (NAT) [2] [3] [4] or antisense oligonucleotide, [5] is a single stranded RNA that is complementary to a protein coding messenger RNA (mRNA) with which it hybridizes, and thereby blocks its translation into protein.
OTvCcg.