Cholesterol-linked drug boosts gene-targeting potency in early findings
Originally written by Institute of Science Tokyo and published in phys.org on October 12, 2026 edited by Swati Mestri , reviewed by Robert Egan Cholesterol-conjugated PMO/RNA heteroduplex oligonucleotide (Chol-HDO[PMO]) achieves high efficacy and high safety, representing a powerful new platform for treating diseases of the central nervous system. Credit: Institute of Science Tokyo Nucleic acid therapeutics , such as antisense oligonucleotides (ASOs) , have emerged as promising platforms for treating a variety of previously hard-to-treat diseases , including neurological diseases. Over the past decade, ASO drugs have been approved worldwide for hereditary amyotrophic lateral sclerosis and spinal muscular atrophy. These drugs are administered directly into the cerebrospinal fluid (CSF) via lumbar puncture, a method known as intrathecal administration, enabling them to reach the brain and spinal cord. Artículo original