• Challenge 79 for World Duchenne Awareness Day
  • About Us
    • Our vision
    • Our Strategy
    • What we do
    • Our Impact
    • Our team
    • Work For Us
    • Volunteer for us
    • Volunteer
    • The DMD Registry
    • Action Duchenne Policies
  • Get Support
    • Mental Health Awareness Week 2025
    • Science on Tour
    • Support Calendar – What’s On
    • Support for you and your family
      • Time Out – A Space for Mums
      • Dads Against Duchenne
      • Grandparents Together
      • Open Space
      • Group Counselling Programme
    • Recently diagnosed
    • Connect with others
    • Support for 8-14 yrs ‘Turning Point’
    • Support for 14-25 yrs ‘Yes I Can’
    • Schools
    • Siblings
    • End of Life and Bereavement
  • AD Annual International Conference
    • SAVE THE DATE for the Action Duchenne Annual International Conference 2025
    • Highlights from the Annual Action Duchenne Annual International 2024
    • Annual International Conference 2023 Video Recordings
    • Annual International Conference 2022 Recordings
      • Adults with Duchenne
      • Growing up with Duchenne
      • The Duchenne Journey
      • What is new in Duchenne research?
  • News, Webinars and Blogs
    • News
    • Webinar Series 2025
      • Webinar Series 2025
      • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
      • Signs and Symptoms of Duchenne Muscular Dystrophy
      • Diagnosis of Duchenne Muscular Dystrophy
      • Crucial Genetic Terminology
      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
    • Blogs
  • Support Us
    • Help Make a Life Beyond Duchenne Possible – Every Month
    • Fundraising Events and Challenges
    • Take on a challenge for Duchenne
    • Shop
  •  0 items - Free
  • Menu
  • Skip to right header navigation
  • Skip to main content
  • Skip to secondary navigation
  • Skip to primary sidebar
  • Skip to footer

Before Header

  • BECOME A MEMBER
  • SHOP
  • My account
  •  0 items - Free

Action Duchenne

Header Right

  • Challenge 79 for World Duchenne Awareness Day
  • About Us
    • Our vision
    • Our Strategy
    • What we do
    • Our Impact
    • Our team
    • Work For Us
    • Volunteer for us
    • Volunteer
    • The DMD Registry
    • Action Duchenne Policies
  • Get Support
    • Mental Health Awareness Week 2025
    • Science on Tour
    • Support Calendar – What’s On
    • Support for you and your family
      • Time Out – A Space for Mums
      • Dads Against Duchenne
      • Grandparents Together
      • Open Space
      • Group Counselling Programme
    • Recently diagnosed
    • Connect with others
    • Support for 8-14 yrs ‘Turning Point’
    • Support for 14-25 yrs ‘Yes I Can’
    • Schools
    • Siblings
    • End of Life and Bereavement
  • AD Annual International Conference
    • SAVE THE DATE for the Action Duchenne Annual International Conference 2025
    • Highlights from the Annual Action Duchenne Annual International 2024
    • Annual International Conference 2023 Video Recordings
    • Annual International Conference 2022 Recordings
      • Adults with Duchenne
      • Growing up with Duchenne
      • The Duchenne Journey
      • What is new in Duchenne research?
  • News, Webinars and Blogs
    • News
    • Webinar Series 2025
      • Webinar Series 2025
      • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
      • Signs and Symptoms of Duchenne Muscular Dystrophy
      • Diagnosis of Duchenne Muscular Dystrophy
      • Crucial Genetic Terminology
      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
    • Blogs
  • Support Us
    • Help Make a Life Beyond Duchenne Possible – Every Month
    • Fundraising Events and Challenges
    • Take on a challenge for Duchenne
    • Shop
  • What is Duchenne?
  • Genetics explained
  • Being a female carrier
  • Clinical trials
  • Potential therapies
  • Glossary of research terms
You are here: Home / What is Duchenne? / Duchenne Explained / Glossary of research terms / Stem cell therapy

Stem cell therapy

Stem cell research may lead to new treatments for Duchenne muscular dystrophy. Normally, muscle stem cells called satellite cells, respond to damage to quickly repair muscle tissue. In Duchenne, these satellite cells cannot respond well to damage and since the body cannot make new satellite cells, these soon become depleted. Stem cell therapy aims to repair a young person’s stem cells before using them to repair and replace their own damaged muscles. Stem cells may help by generating muscle fibres to replace those damaged ones or by helping to reduce the widespread inflammation in the muscle.

So far studies for effectiveness and safety have only been conducted in a mouse model of Duchenne, so this work is still at an early stage. Ongoing research is looking into transplanting dystrophin-producing cells into patients either from healthy donor cells or from genetically modified cells from the patient. A team of scientists are optimistic about the use of CRISPR-Cas9 gene editing to remove the faulty sequence in the dystrophin gene and replace it with a correct sequence. This is done using muscle cells from mdx mice which are reverted to a stem-cell like state and then manipulated to become muscle cells, which are hoped to generate healthy muscle cells when returned to the donor mice. Mark Hamrick, PhD, a scientist involved in this project is optimistic that these modified stem cells have the potential to ‘go back in and rebuild everything’.

Work with stem cells is at an early stage but it has the potential to avoid the problems of immune rejection by treating patients with their own genetically modified and repaired stem cells, instead of those of others.

Share this:

Primary Sidebar

From our community

The Heart of Care

The Heart of Care We have had some key reminders of what we are working towards as a charity over the last few weeks. Volunteer’s Week (2nd – 8th June) was a chance to thank all of the people who give up their time and expertise for Action Duchenne. From the team of trustees, those …

Mental Health Awareness Week: Alex’s Journal

Written By Alex Berbank 15 Minutes a Day I’ve really enjoyed getting into the frame of mind to look at my mental health. Before this week I wasn’t quite sure what to expect. My ideas of what looking at my own mental health would look like weren’t accurate. I thought there would be more softly …

Mental Health Awareness Week: Alex’s Journal

Mental Health Awareness Week Journal, Written by Alex Berbank Watch Alex’s Vlog for Mental Health Awareness Week Thursday’s Update: Meditation and Mindfulness So, to this point this week has been great. I’ve actually taken to the focus on my mental health and self improvement thing quite well and I hope I’ll keep these new ideas …

Footer

Action Duchenne
Wellesley House
Duke of Wellington Avenue Royal Arsenal
London
SE18 6SS

07535 498 506
info@actionduchenne.org 

 

 

 

 

 

 

 

 

Subscribe to our mailing list

Do you consent to receiving regular email updates? *
Email Format
  • Accessibility
  • Privacy Policy
  • Terms & Conditions

© Action Duchenne - Registered Charity No 1101971 - Scottish Charity No SC043852

Like most websites we use cookies to deliver a personalised service. To use the website as intended please accept cookies.
Privacy & Cookies Policy

Privacy Overview

This website uses cookies to improve your experience while you navigate through the website. Out of these, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website. We also use third-party cookies that help us analyze and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these cookies. But opting out of some of these cookies may affect your browsing experience.
Necessary
Always Enabled
Necessary cookies are absolutely essential for the website to function properly. This category only includes cookies that ensures basic functionalities and security features of the website. These cookies do not store any personal information.
Non-necessary
Any cookies that may not be particularly necessary for the website to function and is used specifically to collect user personal data via analytics, ads, other embedded contents are termed as non-necessary cookies. It is mandatory to procure user consent prior to running these cookies on your website.
SAVE & ACCEPT