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.

Thank you

Group of Big Walk participants wearing orange t shirts and walking in a line through countryside

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?

Portrait of Professor Azzouz in a lab at SITRAN

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.

Click to watch video about the Sheffield genetic disease research team

Discover more about the research

The research team tell us what gene therapy is and how it could treat millions of people with rare diseases and other conditions.

Who you'll help

Robbie Edwards with her family, Robbie has leg braces to help with her SPG47

Robbie's story

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.

Brooks' story

There are over 7000 rare diseases with no cure. But from their home in St Louis, USA - the Ward family discovered that Sheffield was investigating a treatment for their son's rare condition.