Genetic Disease Research appeal
From rare conditions like COL4A1 to hearing loss and inherited dementia. With your help, the University of Sheffield will use gene therapies to save lives and rewrite futures.
Will you help?
Your kindness today will help Sheffield develop gene therapy treatments for rare and inherited diseases. You'll bring hope and comfort to families coping with devastating conditions.Donate now
Who you'll help
SPG47 is a painful genetic disease that affects Robbie's speech, movement and learning. But Sheffield research is giving this family hope that there may be a cure.
The power of gene therapy
It's not just rare diseases that are set to benefit from gene therapy. We're invesigating treatments for dementia, MND, hearing loss and kidney disease.
A proven treatment for genetic diseases
Genetic disease research is already saving lives
The team behind Sheffield's gene therapy research has already shown it works. Thanks to a revolutionary new treatment, babies with Spinal Muscular Atrophy now have full futures ahead of them.Discover the potential of gene therapy
Fundraising for the appeal
The Big Walk 2022
Find out more about the Big Walk 2022 - raising money for Genetic Disease Research. We're proud to have over 400 people taking part in the event!
Donate to the Big Walk team
Make a gift to support our Big Walkers as they tackle a challenging 30km or 50km hike through the Peak District. Click to see how much the team has raised towards their target and find out more about the people taking part.