haematology

Gene editing can switch on foetal haemoglobin

Australian-led research has demonstrated the possibility of fighting one genetic mutation with a second ‘beneficial mutation’ to overcome anaemia associated with haemoglobinopathies. The research showed CRISPR-mediated genome editing could be used to introduce the genetic variant British-198 associated with Hereditary Persistence of Fetal Hemoglobin (HPFH) into human umbilical cord blood-derived erythroid progenitor (HUDEP-2) cell lines. The substitution led ...

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