Takeaways from AP’s report on access to gene therapies for rare diseases
Associated PressJun 13
UK Clears Gene-Editing Therapy for Rare Blood Disorder
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Above: An NHS sign sits on the outside of Guy's and St Thomas' Hospital on January 3, 2018 in London, England. Image copyright: Jack Taylor/Stringer/Getty Images News via Getty Images
The Facts
The UK's National Health Service (NHS) has been cleared to use gene-editing therapy to treat the blood disorder beta thalassaemia.
The therapy, termed Casgevy, may help the estimated 2.3k people in the UK with thalassaemia, a rare group of inherited disorders in which the body produces too little hemoglobin, stunting its ability to carry oxygen.
The Spin
Narrative A
Gene-editing therapy will revolutionize thalassemia treatment with the UK’s approval of the groundbreaking Casgevy therapy. It corrects the faulty gene responsible for thalassemia, offering a potential lifelong cure. Previously, the only permanent solution was a bone marrow transplant, requiring a closely matched donor. Casgevy, instead, uses the patient’s own stem cells to produce healthy hemoglobin, drastically reducing the need for regular blood transfusions.
Narrative B
As CRISPR receives an outpouring of positive coverage due to its expected ability to cure diseases, it's important not to forget the negative side of the gene-editing industry. Gene-editing, if left unchecked, will undoubtedly go beyond fixing blood diseases and lead to a world where all differences among humans are artificially eradicated. We shouldn't allow the medical industry to play God when it comes to how we're born and how we develop.
