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Visit the charity's profileAlthough individually uncommon, collectively it's estimated that rare diseases may affect up to 10% of the world's population. In the UK alone, 1 in 25 babies are born with a genetic disorder. Sheffield's research will change the lives of families who are desperately seeking a treatment for their loved-one.
Sheffield's scientists have discovered a way to engineer bespoke genes to replace or silence a faulty one. In the form of a safe viral vector these gene therapies will be manufactured in Sheffield and delivered to patients around the world.
The Sheffield team behind this research has already helped to develop gene therapy treatments for babies with Spinal Muscular Atrophy (SMA).
Babies suffering from SMA previously had a life expectancy of just two years, and were often left paralysed and unable to breathe on their own. After a single treatment, babies are now able to breathe without a ventilator and learn to walk. Some have even been able to start school.
By supporting this research you'll help Sheffield discover ways to treat even more genetic diseases. From genetic forms of Motor Neurone Disease to rare diseases such as COL4A1.
COL4A1 can cause strokes, spasms and heart defects in babies and can be detected as early as a 20 week pregnancy scan.
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