Genetic Disease Research appeal

From rare conditions like COL4A1 to hearing loss and inherited dementia. With your help, the �־�� 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.

Donate now

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.

Finding cures for genetic conditions

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.

Treatments for incurable diseases

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.

Hope for families with rare diseases

A proven treatment for genetic diseases

Babies Grace and Sebastian smiling at the camera

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