Recommended

Discover how to overcome low affinity or poor TCR yields that are slowing you down in this upcoming webinar – register your details today
WEBINAR | Discover how advances can expand the possibilities for drug discovery, from target validation to preclinical development
Facing roadblocks in obesity drug discovery? Discover how integrated, validated strategies are helping teams accelerate development and reduce risk – register your details now
Unlock the science, strategy and real-world impact behind the next generation of precision therapies – access our new brand report now >>
Safety data is no longer just a regulatory checkbox – it’s a strategic advantage. Find out more in our upcoming webinar, register your details today
Explore how AI is reshaping early drug discovery, from target identification and compound screening to predictive modelling and trial design – register now!
news

Researchers find DNA therapy could treat patients with DMD

A study has revealed that using DNA-like molecules to repair gene mutations in models could act as a successful therapy for patients.

DNA-like molecule for DMD treatment

Researchers have used a promising new therapeutic to treat Duchenne muscular dystrophy (DMD) in human muscle cells and mice models. According to the researchers, their treatment could aid as many as 47 percent of patients with the condition.

The study, conducted by the University of Alberta, US, investigated whether using a group of DNA-like molecules would result in regrowth of a protein called dystrophin, which supports muscle strength. Patients with DMD often severely lack this protein.

“In muscle, if there is no dystrophin there is no support of muscle membrane and the muscle cells will become easily damaged or destroyed,” said Toshifumi Yokota, a professor of medical genetics at the university. “Our DNA-like molecules restore the production of dystrophin so it can support the muscle cell membrane.”

 

Reserve your FREE place

 


Reduce preclinical failures with smarter off-target profiling

24 September 2025 | 15:00PM BST | FREE Webinar

Join this webinar to hear from Dr Emilie Desfosses as she shares insights into how in vitro and in silico methods can support more informed, human-relevant safety decisions -especially as ethical and regulatory changes continue to reshape preclinical research.

What you’ll learn:

  • Approaches for prioritizing follow-up studies and refining risk mitigation strategies
  • How to interpret hit profiles from binding and functional assays
  • Strategies for identifying organ systems at risk based on target activity modulation
  • How to use visualization tools to assess safety margins and compare compound profiles

Register Now – It’s Free!

 

The team used a mix of DNA-like molecules, known as antisense oligonucleotides, to act in the manner of a stitch to repair a specific gene mutation in patients.

“Our treatment produces a shorter dystrophin protein than the drug being used now. This shorter protein is associated with extremely mild symptoms in some of the muscular dystrophy patients. Some have almost no symptoms at all,” explained Yokota.

The researchers are now working to reduce the number of DNA-like molecules in the mixture to reduce both cost and regulatory hurdles moving forward. They hope to progress the work to a clinical trial in the future.

The results were published in Molecular Therapy.