Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …

Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …

Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure
for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …

Systematic evaluation of the impact of genetic variants is critical for the study and treatment
of human physiology and disease. While specific mutations can be introduced by genome …

The clinical success of CRISPR therapies hinges on the safety and efficacy of Cas proteins.
The Cas9 from Francisella novicida (FnCas9) is highly precise, with a negligible affinity for …

Accurately quantifying the functional consequences of noncoding mosaic variants requires
the pairing of DNA sequences with both accessible and closed chromatin architectures …

Thalassemia syndromes are among the commonest monogenic disorders and represent a
substantial health burden worldwide. Here the authors have described globin genes …

After many years of painstaking research, the potential of gene therapy to cure severe β-
thalassemia is now becoming evident through recent clinical trials. Therapeutic protocols …

Sickle cell disease (SCD) is one of the most common hemoglobinopathies. Due to its high
prevalence, with about 20 million affected individuals worldwide, the development of novel …

Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …