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UK Child's Life Transformed by Gene Editing
29 Jan
Summary
- A boy received pioneering gene-editing therapy for thalassaemia.
- The £1.65 million treatment uses CRISPR technology to edit faulty genes.
- He no longer needs monthly blood transfusions after successful therapy.
A 13-year-old boy from Oldham has had his life transformed by a groundbreaking gene-editing therapy, becoming one of the first in the UK to receive the treatment. Adam Rehman was born with beta-thalassaemia, a severe inherited blood disorder that previously necessitated monthly blood transfusions.
The one-time gene therapy, known as Casgevy (exa-cel), utilizes CRISPR gene-editing technology to correct faulty genes within the patient's own stem cells. This scientific breakthrough, recognized with a Nobel Prize, has now offered Adam a cure.
Adam received the specialized treatment in November 2024, a process involving the collection of his stem cells, gene editing in a lab, and reinfusion. This therapy provides a crucial option for patients who could benefit from a stem cell transplant but lack a suitable donor.
Recently, Adam returned to the Royal Manchester Children's Hospital to express his gratitude to Clair Baron, a specialist nurse who played a vital role in his treatment. He shared how good it felt to regain his freedom and no longer be confined to hospital.
His father echoed these sentiments, expressing deep gratitude for Nurse Baron's involvement. The medical team highlighted the life-changing impact of such gene therapies, emphasizing the privilege of supporting patients on their journey to recovery.
