Ariya Bio

Reactivating foetal haemoglobin in B-Haemoglobinopathy patients

Haemoglobinopathies are one of the most widespread genetic diseases worldwide. In these hereditary diseases, the structure, function or formation of haemoglobin is disturbed, so that oxygen transport in the blood is restricted. Today, apart from stem cell transplantation (which is not always successful), there is no effective therapy for the disease. Mandy Boontanrart wants to change this. Her therapeutic approach is based on observations that haemoglobinopathy patients with elevated foetal haemoglobin have milder disease symptoms than most others, in whom it decreases sharply after birth. Mandy Boontanrart uses the CRISPR/Cas9 gene editing method to reactivate foetal haemoglobin.

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