Understanding and curing sickle cell disease
University of East London University of East London
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 Published On Apr 9, 2024

Sickle Cell Disease is an inherited blood disorder, which occurs predominantly in people of African and African Caribbean origin, that due to a genetic alteration (mutation) in the gene that produces the oxygen carrying protein haemoglobin. The condition leads to symptoms of anaemia, severe pain an increased risk of infections, organ failure and early death. The only cure is bone marrow transplantation but is fraught with difficulties.

Treatment consists of regular blood transfusion along with drugs, antibiotics and painkillers. However, these are all fraught with problems. Recently UK medical regulators approved "first-of-its-kind" gene-editing treatment called Casgevy, which uses CRISPR DNA technology, to treat sickle cell, with clinical trials showing positive results. what is CRISPR technology and could this gene therapy approach be the ultimate cure for sickle cell disease?

The video with Professor Winston Morgan of UEL will explain what sickle cell disease is, how you inherit it, current treatment, how the new treatment works and the safety concerns.

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