The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Abstract excerpt

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 diseases. While SMA specifically affects spinal motor neurons, other...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Neurogenetic and Muscular Disorders Research
• Share to RNA Interference and Gene Delivery
• Share to RNA Research and Splicing