Research & Development World

  • R&D World Home
  • Topics
    • Aerospace
    • Automotive
    • Biotech
    • Careers
    • Chemistry
    • Environment
    • Energy
    • Life Science
    • Material Science
    • R&D Management
    • Physics
  • Technology
    • 3D Printing
    • A.I./Robotics
    • Software
    • Battery Technology
    • Controlled Environments
      • Cleanrooms
      • Graphene
      • Lasers
      • Regulations/Standards
      • Sensors
    • Imaging
    • Nanotechnology
    • Scientific Computing
      • Big Data
      • HPC/Supercomputing
      • Informatics
      • Security
    • Semiconductors
  • R&D Market Pulse
  • R&D 100
    • Call for Nominations: The 2025 R&D 100 Awards
    • R&D 100 Awards Event
    • R&D 100 Submissions
    • Winner Archive
    • Explore the 2024 R&D 100 award winners and finalists
  • Resources
    • Research Reports
    • Digital Issues
    • R&D Index
    • Subscribe
    • Video
    • Webinars
  • Global Funding Forecast
  • Top Labs
  • Advertise
  • SUBSCRIBE

Antisense Oligonucleotides May Treat Myotonic Dystrophy

By R&D Editors | February 28, 2012

Antisense oligonucleotides—short segments of genetic material designed to target specific areas of a gene or chromosome—that activated an enzyme to “chew up” toxic RNA may point the way to a treatment for myotonic dystrophy, says researchers from Baylor College of Medicine and Isis Pharmaceuticals Inc.


“This is a proof-of-principle therapy that is effective in cell culture and mice,” says Thomas A. Cooper, MD, professor of pathology and immunology and molecular and cellular biology at Baylor College of Medicine. “The treatment will have to be refined to deliver systemically in people with myotonic dystrophy.”


Myotonic dystrophy occurs because of a mutation that causes numerous repeats of three letters of the genetic code (CTG) in a gene called DMPK. RNA is made as a step in the cell’s production of the protein associated with the gene. The messenger RNA that is produced from the mutated gene also contains the abnormal long repeats that cause the RNA to accumulate in the cell’s nucleus. There it sequesters and blocks the function of a Muscleblind-like 1 protein and activates another protein, CELF1. The proteins antagonize one another and the result is abnormal expression of proteins from many other genes in adult tissues, resulting in disease.


To counteract this, Cooper and his colleagues created gapmers (antisense oligonucleotides), that are strands of genetic material that seek out portions of the abnormal RNA repeats and target an RNase H enzyme to the toxic RNA, causing its degradation. They also showed that combining the gapmers with other antisense oligonucleotides that help released the sequestered Muscleblind-like1 can enhance the effect.


“It worked in cultures of cells with the expanded repeats and in mice that model myotonic dystrophy,” says Cooper. “We did it in skeletal muscle first because we can inject the material directly into the muscle.”


The researchers saw some muscle damage and inflammation in the animals they treated. Using the treatment in people will require more fine-tuning, says Cooper. He would like to be able to give the therapy systemically rather than directly into the muscle.


The research was reported in the Proceedings of the National Academy of Sciences.


Release Date: Feb. 27, 2012
Source: Baylor College of Medicine

Related Articles Read More >

Eli Lilly facility
9 R&D developments this week: Lilly builds major R&D center, Stratolaunch tests hypersonic craft, IBM chief urges AI R&D funding
professional photo of wooly mammoth in nature --ar 2:1 --personalize sq85hce --v 6.1 Job ID: 47185eaa-b213-4624-8bee-44f9e882feaa
Why science ethicists are sounding skepticism and alarm on ‘de-extinction’
ALAFIA system speeds complex molecular simulations for University of Miami drug research
3d rendered illustration of the anatomy of a cancer cell
Funding flows to obesity, oncology and immunology: 2024 sales data show where science is paying off
rd newsletter
EXPAND YOUR KNOWLEDGE AND STAY CONNECTED
Get the latest info on technologies, trends, and strategies in Research & Development.
RD 25 Power Index

R&D World Digital Issues

Fall 2024 issue

Browse the most current issue of R&D World and back issues in an easy to use high quality format. Clip, share and download with the leading R&D magazine today.

Research & Development World
  • Subscribe to R&D World Magazine
  • Enews Sign Up
  • Contact Us
  • About Us
  • Drug Discovery & Development
  • Pharmaceutical Processing
  • Global Funding Forecast

Copyright © 2025 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search R&D World

  • R&D World Home
  • Topics
    • Aerospace
    • Automotive
    • Biotech
    • Careers
    • Chemistry
    • Environment
    • Energy
    • Life Science
    • Material Science
    • R&D Management
    • Physics
  • Technology
    • 3D Printing
    • A.I./Robotics
    • Software
    • Battery Technology
    • Controlled Environments
      • Cleanrooms
      • Graphene
      • Lasers
      • Regulations/Standards
      • Sensors
    • Imaging
    • Nanotechnology
    • Scientific Computing
      • Big Data
      • HPC/Supercomputing
      • Informatics
      • Security
    • Semiconductors
  • R&D Market Pulse
  • R&D 100
    • Call for Nominations: The 2025 R&D 100 Awards
    • R&D 100 Awards Event
    • R&D 100 Submissions
    • Winner Archive
    • Explore the 2024 R&D 100 award winners and finalists
  • Resources
    • Research Reports
    • Digital Issues
    • R&D Index
    • Subscribe
    • Video
    • Webinars
  • Global Funding Forecast
  • Top Labs
  • Advertise
  • SUBSCRIBE