Article

Advances in Gene Therapy for Inherited Haemoglobinopathies

2025-11-28

Abstract excerpt

Haemoglobinopathies, including β-thalassaemia and sickle cell disease (SCD), are among the most common monogenic disorders worldwide and remain major causes of morbidity and early mortality. Historically, management of these life-altering diseases has relied on supportive treatment and symptom management and although these treatments reduce symptoms and ease disease burden, they do not correct the underlying genet...

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Literature Corpus work: 66b693e7-aea2-5bf0-9f35-32ef0a84d25a
DOI: 10.20944/preprints202511.2257.v1

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