Therapeutic Antisense Targeting of Huntingtin

摘要

Antisense oligonucleotides (ASOs) are a relatively new therapeutic entity that utilizes short chemically modified strands of DNA in targeted interactions with RNA to modulate the type or amount of resultant protein. This brief review summarizes the preclinical, translational, and early clinical development of an ASO designed to reduce the production of the disease-causing protein in Huntington's disease, an inherited neurodegenerative disease.