Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe
monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a …

… Author links open overlay panel Amanda M. Li MD * 1 , George E Hucks * 2 , Amanda M.
Dinofia MD * 3 , Alix E. Seif MD 4 , David T Teachey MD 5 , Diane Baniewicz * 3 , Colleen …

Background Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to
reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short …

White matter injury (WMI) is the characteristic pattern of brain injury detected on magnetic
resonance imaging in the premature newborn. Focal noncystic WMI is increasingly recognized …

10.7554/eLife.28672.001 Oriented cell division is one mechanism progenitor cells use
during development and to maintain tissue homeostasis. Common to most cell types is the …

Glycogen storage disease (GSD) type 1 is a rare autosomal recessive inherited condition.
The 1b subtype comprises the minority of cases, with an estimated prevalence of 1 in 500,000 …

Hippocampal slices obtained from C57BL/6 mice (3–25 mo) were used to investigate the
effects of aging on excitatory postsynaptic potentials (EPSPs) elicited in dentate gyrus with …

Introduction: CAR T cells in B-ALL have recently focused on the dual targeting of CD19 and
CD22 to enhance long term remissions and prevent antigen negative recurrence that is …

BACKGROUND The cure rate for childhood intracranial ependymoma is approximately 70%
in the setting of a gross total resection followed by radiation, but management remains …

INTRODUCTION Children with Down Syndrome (DS) and B-lymphoblastic leukemia (B-ALL)
are at an increased risk of both relapse and treatment-related mortality, compared to those …