Gene therapy is lifesaving, it’s revolutionary! It’s changed our lives. It’s given my son mobility that he’d lost. The possibilities are unknown but exciting. Knowing that Sebastian will not deteriorate and regress brings us such peace – in our hearts and minds. It’s monumental. You never know who will be impacted by a rare disease. It affects all those connected to Sebastian and me. When you donate, you’re helping to fund research into life-changing, disease-altering medicine. You’re not only helping the individual with the rare disease, you’re changing the life of an entire network of people whose lives will be forever changed by a diagnosis. It’s so much more than just a donation. I’m incredibly proud that my University is leading this field of research – I may have left the University of Sheffield but it never left me.” Dharmisha Stezaly BA Accounting and Financial Management (Class of 2012) and mother to Sebastian who received gene therapy for SMA type 1.
Scientists can then engineer therapeutic genes and introduce them into the virus. The viral vectors are used as carriers to deliver the therapeutic genes into a patient’s cells. As it’s a type of virus, the treatment spreads quickly throughout the body but the virus does not replicate or cause any damage. This type of gene therapy can repair or switch off a problematic gene or introduce a missing gene into a patient’s cells, helping to delay, stop or even reverse the effects of a disease.
Bringing hope to families with genetic diseases Professor Mimoun Azzouz has already shown that gene therapy can work, by making several discoveries in this discipline. Babies with spinal muscular atrophy (SMA) type 1 are now recovering from this childhood form of MND, after just one dose of the gene therapy drug Zolgensma©. It’s administered by drip and only takes one hour. And it’s safe for babies as young as two weeks old. Now, the Sheffield community is coming together to help even more people with genetic diseases. Hundreds of alumni, staff and friends of Sheffield have been donating and taking on fundraising challenges. Together they’re helping to fund crucial equipment that will accelerate research towards clinical trials.
Why Sheffield is uniquely placed to lead the gene therapy revolution ›› Research programmes that unravel the mysteries of disease – from neurological conditions like MND to age-related hearing loss.
›› A recognised global player in gene therapy – we’re leading a prestigious consortium of European universities and major pharmaceutical companies.
›› Experts in clinical trials and translational medicine, the University of Sheffield is experienced at taking science from the lab and turning it into real treatments.
›› Selected as one of only three gene therapy hubs in the UK, the new Gene Therapy Innovation and Manufacturing Centre (GTIMC) will expand research capacity and manufacture treatments at scale.
Will you help Sheffield find treatments for people with life-limiting conditions? For families coping with devastating genetic disorders, your support will bring hope and comfort. And you’ll help put your University at the heart of a treatment revolution. Call today: 0114 222 1071 Complete and return the form enclosed with your magazine.
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DONATE NOW sheffield.ac.uk/giving/gdr
2022/2023 | YOUR UNIVERSITY 17