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haemoglobinopathy (hemoglobinopathy)

Autologous therapies for hemoglobinopathies

Hemoglobinopathies are a group of inherited blood disorders where hemoglobin, the oxygen-carrying molecule in red blood cells, is defective: abnormal hemoglobin structure or production results from deletions or point mutations in the alpha or beta-globin genes. The most frequent hemoglobinopathy is sickle cell anemia (SCA), which results from mutations in the beta-globin (HBB) gene that affect the structure and function of the hemoglobin protein. In thalassemia, a mutation in the alpha or beta chain leads to reduced production of hemoglobin.

Autologous therapies for hemoglobinopathies. Autologous CD34+ HSPC is edited with CRISPR/Cas9 to repair mutations in the beta-globin gene associated with beta-thalassemia and SCA. “Created with BioRender.com.

The only curative treatments for hemoglobinopathies have relied on allogeneic transplantation of hematopoietic and progenitor cells (HSPC). However, advances in gene editing technologies, such as CRISPR/Cas9 tools, have provided new alternative curative treatments for hemoglobinopathies.

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