Friedreich's ataxia (FRDA) is an inherited, multisystemic disorder predominantly caused by
GAA hyper expansion in intron 1 of frataxin (FXN) gene. This expansion mutation …

Multiple sclerosis (MS) is a heterogeneous disease of the central nervous system that is
governed by neural tissue loss and dystrophy during its progressive phase, with complex …

Hematopoietic stem cell transplantation can deliver therapeutic proteins to the central
nervous system (CNS) through transplant-derived microglia-like cells. However, current …

Autologous hematopoietic stem cell transplantation using genome-edited cells can become
a definitive therapy for hematological and non-hematological disorders with neurological …

Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) has shown clear neurological
benefit in rare diseases, which is achieved through the engraftment of genetically modified …

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) with CCR5–donor cells is
the only treatment known to cure HIV-1 in patients with underlying malignancy. This is likely …

Hematopoietic stem cell transplantation is a well-known treatment for hematologic
malignancies, wherein nascent stem cells provide regenerating marrow and immunotherapy …

Macrophages orchestrate tissue immunity from the initiation and resolution of antimicrobial
immune responses to the repair of damaged tissue. Murine studies demonstrate that tissue …

Pompe disease is a neuromuscular disorder caused by a deficiency of the lysosomal
enzyme acid alpha-glucosidase (GAA), hydrolyzing glycogen to glucose. Pathological …

While new immunotherapies have revolutionized the field of oncology, they have been
limited by their inability to distinguish between cancerous cells and healthy HSPCs. Work by …