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.
You're bringing hope to so many people
Thanks to the support of almost 5,000 donors so far, Sheffield researchers are getting one step closer to creating new treatments for genetic diseases.Follow the progress of the GTIMC
Could you help the team do even more?
Make a gift
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.
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 and hearing loss.
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