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

Simple, efficient and well-tolerated delivery of CRISPR genome editing systems into primary
cells remains a major challenge. Here we describe an engineered Peptide-Assisted …

Gene therapy has shown significant potential in treating various diseases, particularly
inherited blood disorders such as hemophilia, sickle cell disease, and thalassemia …

The fetal-to-adult switch in hemoglobin production is a model of developmental gene control
with relevance to the treatment of hemoglobinopathies. The expression of transcription factor …

During development, erythroid cells are produced through at least 2 distinct hematopoietic
waves (primitive and definitive), generating erythroblasts with different functional …

Fetal Hemoglobin Regulation in Beta-Thalassemia Page 1 Fetal hemoglobin regulation in
beta-thalassemia 1 2 Henry Y. Lu, PhD1,2,3, Stuart H. Orkin, MD1,2,4,5, Vijay G. Sankaran …

Genome-wide analysis of transcription factors and epigenomic features is instrumental to
shed light on DNA-templated regulatory processes such as transcription, cellular …

In humans, specific aberrations in β-globin results in sickle cell disease and β-thalassemia,
symptoms of which can be ameliorated by increased expression of fetal globin (HbF). Two …

The switch from fetal hemoglobin (HbF) to adult hemoglobin (HbA) is a paradigm for
developmental gene expression control with relevance to sickle cell disease and β …

The CRISPR/Cas9 technique is easily programmable, fast, more powerful, and efficient at
generating a mutation compared to previous gene therapy methods. β-thalassemia is the …