Cholesterol-linked drug boosts gene-targeting potency in early findings
Researchers have developed a cholesterol-conjugated oligonucleotide platform to improve the delivery and safety of gene-targeting therapies for neurological diseases. This new method aims to overcome the toxicity issues associated with current antisense oligonucleotide treatments.
Why it matters
This advancement could lead to safer and more effective treatments for hereditary neurological conditions, reducing the side effects currently seen in spinal and brain therapies.
edited by Swati Mestri , reviewed by Robert Egan
This article has been reviewed according to Science X's editorial process and policies . Editors have highlighted the following attributes while ensuring the content's credibility:
Add to Preferred Sources 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.
Get smarter about the news
Sign up free for a feed built around what you actually care about, Dive Deeper research on any story, and the full text of every article.
Create free accountAlready have an account? Sign in