Sisters Liz Gill and Sue Burrell have both had their lives turned around by gene silencing.
Before treatment, Liz remembers the trauma of living in "total pain" and, at its worst, she spent two years paralysed in hospital. Younger sister Sue says she "lost it all overnight" when she was suddenly in and out of hospital, made redundant and did know whether her partner would stick with her .Both became used to taking potent opioid painkillers on a daily basis. But even morphine could not block the pain during a severe attack that needed hospital treatment.
Both had been taking the therapy as part of a clinical trial and are still getting monthly injections."You're not dependent on opiate-based pain relief and that leads to things like being able to succeed in a job and being able to buy your own home."Sue said the therapy had transformed her life: "[You're] able to do things that you couldn't do before, being able to be a mother better, being able to be a wife better… to just live life.
However, acute intermittent porphyria is rare. Only around 17 people are diagnosed in the UK each year. "[But] if we can control genes and switch them on and off when we want to, then almost anything is possible in terms of treating diseases including Alzheimer's and cancer and everything else," Prof Rees said.
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