Cookie use on MRCVSonline
We use cookies to ensure that we give you the best experience on our website. If you continue without changing your settings, we will assume that you are happy to receive all cookies.
If you would like to forward this story on to a friend, simply fill in the form below and click send.

Your friend's email:
Your email:
Your name:
 
 
Send Cancel

Gene study offers hope for treating fatal illness in children and dogs
Using SingleCut CRISPR gene editing, they managed to modify the dogs’ own DNA so they began producing dystrophin again.

Doctors and vets team up to study Duchenne muscular dystrophy

Vets and medics say they have made a significant breakthrough in the search for a treatment for Duchenne muscular dystrophy (DMD) - a fatal disease that affects children and dogs.

Scientists from the Royal Veterinary College and UT Southwestern Medical Centre used gene editing techniques in dogs, to restore the production of a protein that is absent in DMD patients.

DMD is the most common fatal genetic disease in children and it also occurs in many dog breeds. Patients with the disease produce almost no dystrophin, a protein that helps to protect muscle fibres and the heart when they contract. As a result, the patient’s muscles are damaged, leading to an inability to walk, skeletal deformities and heart failure.

The team said they may have come up with a single intravenous injection to treat the most common genetic mutations.

A naturally occurring mutation in the dystrophin gene was found in a dog that was being treated at the RVC’s Small Animal Referral Hospital, and the team are now working with the dog’s relatives. Using SingleCut CRISPR gene editing, they managed to modify the dogs’ own DNA so they began producing dystrophin again.

In a dog that received the highest dose, the cardiac muscles restored dystrophin protein to levels up to 92 per cent of the normal amount after treatment.

Scientists believe this is the first time this type of gene editing therapy has been used after birth in an animal larger than a rodent. It is hoped there will be a human application, after long-term research, efficacy and safety testing.

Richard Piercy, professor of comparative neuromuscular disease at the RVC, said: “There is more work to be done, but with this exciting breakthrough, we feel we are a significant step closer to finding an effective treatment.
 
“A great many genetic diseases affect humans, dogs and other species - many, like DMD - with tragic consequences and this form of gene editing treatment might well be applicable in many of them. We’re hopeful that we’re paving the way for future research into treating some of the most serious genetic conditions that affect us today.”

Preliminary findings have been published in the journal Science.

Image © RVC
 

Become a member or log in to add this story to your CPD history

Veterinary Evidence Student Awards winners revealed

News Story 1
 The first winners of the RCVS Knowledge Veterinary Evidence Student Awards have been revealed.

Molly Vasanthakumar scooped first prize for her knowledge summary comparing the ecological impact of woven versus disposable drapes. She found that there is not enough evidence that disposable synthetics reduce the risk of surgical site.

Second prize went to Honoria Brown of the University of Cambridge, for her paper: ‘Can hoof wall temperature and digital pulse pressure be used as sensitive non-invasive diagnostic indicators of acute laminitis onset?’

Edinburgh’s Jacqueline Oi Ping Tong won third prize for critically appraising the evidence for whether a daily probiotic improved clinical outcomes in dogs with idiopathic diarrhoea. The papers have all achieved publication in RCVS Knowledge’s peer-reviewed journal, Veterinary Evidence.  

Click here for more...
News Shorts
Animal Welfare Foundation seeks new trustees

The Animal Welfare Foundation (AWF) seeks three new trustees to help drive the charity’s mission to improve animal welfare through veterinary science, education and debate.

Veterinary and animal welfare professionals from across the UK may apply, particularly those with experience in equine and small animal practice and research management. Trustees must attend at least two meetings a year, as well as the annual AWF Discussion Forum in London.

For more information about the role, visit www.animalwelfarefoundation.org.uk. Applications close at midnight on 13 August 2019.