Scientists say using CRISPR-Cas9 to modify specific subset of stem cells would effectively reduce the costs of gene-editing treatments for blood disorders, while minimizing the risks of off-target ...

We had previously reported the presence of an apparent increase in Type II hexokinase activity in the red cells of a patient homozygous for the hereditary persistence of fetal hemoglobin, and in ...

Besides being a benign genetic condition, the hereditary persistence of fetal hemoglobin could, with some help from CRISPR, lead to a new treatment for common blood disorders, such as sickle cell a ...

The focus of Syros’ drug discovery program in sickle cell disease is to develop an oral medicine to mimic a condition found in a subset of patients, who also inherit a hereditary persistence of ...

CDA IV combines features of hemoglobinopathy, red blood cell (RBC) membrane defects, and hereditary persistence of fetal hemoglobin (HPFH), which are not seen in the other types of CDA. 11, 12 ...

Fetal hemoglobin is naturally shut down after birth but can be reactivated by disrupting the BCL11A gene expression during erythropoiesis. “In β-thalassemia patients with hereditary persistence ...

Editas Medicine’s in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to ...

That technique reawakens the fetal gene and, in at least three patients, has alleviated symptoms of sickle cell disease. The new trial is a gene knock-in: The researchers are using CRISPR-Cas9 to ...

Syros Pharmaceuticals, a leader in the development of medicines that control the expression of genes, today announced that it has discovered and validated a novel fetal hemoglobin repressor ...

Data demonstrate therapeutically relevant editing levels using a clinically validated strategy, supporting its development as a novel, in vivo approach to treating sickle cell disease and beta ...