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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34 + cell therapy to induce fetal hemoglobin for sickle cell disease

2024-05-02

Abstract excerpt

Sickle cell disease (SCD) is a common severe blood disorder, caused by one major point mutation in the HBB gene. Current pharmacotherapies are only partially effective and potentially curative allogeneic hematopoietic stem cell transplantation (HSCT) is associated with immune toxicities. Genome editing of autologous patient hematopoietic stem cells (HSCs) to reactivate fetal hemoglobin (HbF) in erythroid progeny...

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Literature Corpus work: 477a9936-04ad-5350-bd04-334b2549b0af
DOI: 10.1101/2024.04.30.591737

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34 <sup>+</sup> cell therapy to induce fetal hemoglobin for sickle cell disease

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