Editas Medicine Reports Data Supporting Multiple Approaches to Create Treatments for Sickle Cell Disease and Beta-Thalassemia
In experiments, CRISPR/Cas9 caused efficient and reproducible HDR in CD34+ cells with minimal impact on cell viability. In separate studies, CRISPR/Cpf1 efficiently edited at multiple sites, including targets associated with hereditary persistence of fetal hemoglobin (HPFH). These results confirm that Cpf1-directed editing expands the number of genomic sites accessible to develop genome editing medicines.
“We are encouraged by these results demonstrating efficient targeted integration at the beta-hemoglobin locus with CRISPR/Cas9 and results demonstrating efficient on target editing of adult human hematopoietic stem cells with CRISPR/Cpf1. These data together support multiple approaches to creating a superior therapy for the treatment of sickle cell disease and beta-thalassemia,” said
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Source: Editas Medicine, Inc.