Hereditary persistence of fetal hemoglobin is a condition in which levels of HbF persist at levels greater than typically expected (less than 1%). In hereditary persistence of fetal hemoglobin ...

Sinai J. Med. 72, 333–338 (2005). PubMed Google Scholar Sankaran, V.G. et al. Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A. Science 322 ...

Panel C shows naturally occurring variants that cause hereditary persistence of fetal hemoglobin (HPFH). Single-base mutations that lead to increased γ-globin gene expression are indicated.

We found a new (δβ) 0-thalassemia deletion and a rare hereditary persistence of fetal hemoglobin deletion with identical downstream breakpoints. Comparison of the two deletions resulted in the ...

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 ...

who also inherit a hereditary persistence of fetal hemoglobin (HPFH) mutation, in which the gamma-globin gene remains activated after birth. Despite having the mutated adult beta-globin gene that ...

The first medicine based on gene editing, a one-time therapy for sickle cell disease, was just approved in the United States. It’s a big moment for patients with sickle cell disease and for the ...

This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for ...

In 2014, two years after her Nobel Prize-winning invention of CRISPR-Cas9 genome editing, Jennifer Doudna thought the technology was mature enough to tackle a cure for a devastating hereditary ...

Treatment with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or ...