NEJM and Eric Topol highlight multiple trials using Cas12a and base editing to target HBG1 and HBG2 promoters in autologous stem cells for sickle cell disease and transfusion-dependent beta thalassemia, expanding gene-editing options.
3 new trial results for genome editing of sickle cell disease and β-thalassemia @NEJM
In the RUBY study, a Cas12a–guide RNA complex was used to target the promoters of HBG1 and HBG2 in autologous stem cells to treat sickle cell disease.
In the EdiThal study, a Cas12a–guide RNA complex was used to target the promoters of HBG1 and HBG2 in autologous stem cells to treat transfusion-dependent β-thalassemia.
In the BEACON study involving persons with sickle cell disease, adenine base editing was used to target the promoters of HBG1 and HBG2 in autologous hematopoietic stem cells to increase fetal hemoglobin expression.
In the BEACON study involving persons with sickle cell disease, adenine base editing was used to target the promoters of HBG1 and HBG2
In the RUBY study, a Cas12a–guide RNA complex was used to target the promoters of HBG1 and HBG2
In the BEACON study involving persons with sickle cell disease
adenine base editing was used to target the promoters of HBG1 and HBG2
in autologous hematopoietic stem cells
to increase fetal hemoglobin expression.
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