Genetic diseases of blood cells are prime candidates for treatment through ex vivo gene
editing of CD34 + hematopoietic stem/progenitor cells (HSPCs), and a variety of technologies …

We have studied enhancer function in transient and stable expression assays in mammalian
cells by using systems that distinguish expressing from nonexpressing cells. When …

… MILEs placed in upstream promoter regions are able to increase the level of ~-geo
expression in K562 cells when stimulated with zinc (W. Magis and DIK Martin, unpubl. I. The …

In cell senescence, cultured cells cease proliferating and acquire aberrant gene expression
patterns. MicroRNAs (miRNAs) modulate gene expression through translational repression …

Murine erythroleukemia (MEL) cells are a model system to study reorganization of the
eukaryotic nucleus during terminal differentiation. Upon chemical induction, MEL cells undergo …

Background A point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin
subunit of hemoglobin, with resultant anemia and multiorgan damage that typically …

Sickle Cell Disease and ß-thalassemia, which are caused by defective or deficient adult ß-globin
(HBB) respectively, are the most common serious genetic blood diseases in the world. …

A constitutive DNase I-hypersensitive site 5′ of the chicken β-globin locus, termed 5′HS4
or cHS4, has been shown to insulate a promoter from the effect of an upstream enhancer …

Sickle Cell Disease (SCD), one of the world’s most common genetic disorders, causes anemia
and progressive multiorgan damage that typically shortens lifespan by decades; currently …

The mouse metallothionein-I (mMT-I) promoter is activated by the metal response element-binding
transcription factor (MTF), which binds metal response elements (MREs) when …