Importance Sickle cell disease (SCD) is an inherited disorder of hemoglobin, characterized
by formation of long chains of hemoglobin when deoxygenated within capillary beds …

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

Background Sickle cell disease is characterized by the painful recurrence of vaso-occlusive
events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; …

Gene therapy with LentiGlobin for sickle cell disease (bb1111, lovotibeglogene autotemcel)
consists of autologous transplantation of a patient's hematopoietic stem cells transduced …

Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …

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

Platelets are chief cells in hemostasis. Apart from their hemostatic roles, platelets are major
inflammatory effector cells that can influence both innate and adaptive immune responses …

DNA double-strand breaks (DSB) are repaired by multiple distinct pathways, with outcomes
ranging from error-free repair to mutagenesis and genomic loss. DSB-repair pathway cross …

Psychological stress has adverse effects on various human diseases, including those of the
cardiovascular system. However, the mechanisms by which stress influences disease …

Polymerization of deoxygenated hemoglobin S underlies the pathophysiology of sickle cell
disease (SCD). In activating red blood cell pyruvate kinase and glycolysis, mitapivat (AG …