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Monocyte-derived IL-1 and IL-6 are differentially required for cytokine-release syndrome and neurotoxicity due to CAR T cells
A mouse model recapitulating key features of CRS and neurotoxicity is described, offering a therapeutic strategy to tackle neurotoxicity and open new avenues to safer CAR T cell therapies. Expand
HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia.
The data show that genetic manipulation of donor lymphocytes may increase the efficacy and safety of allo-BMT and expand its application to a larger number of patients. Expand
Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
Sustained engraftment of engineered HSCs with differentiation into multiple lineages resulted in increased lymphocyte counts, improved immune functions, and lower toxic metabolites, indicating the safety and efficacy of HSC gene therapy combined with nonmyeloablative conditioning for the treatment of SCID. Expand
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.
Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency and effective protection against infections and improvement in physical development made a normal lifestyle possible. Expand
Gene therapy
IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.
It is shown that it is possible to differentiate in vitro, expand, and gene modify in clinically compliant conditions CD8(+) T(SCM) lymphocytes starting from naive precursors, suggesting self-renewal capacity in a clinically relevant setting. Expand
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
The reconstitution of ARSA activity in the cerebrospinal fluid and the arrested progression of neurodegenerative disease in the three treated patients demonstrate that the transplanted cells, or their progeny, can seed the nervous system and deliver therapeutic levels of active enzyme. Expand
Gene Therapy in Peripheral Blood Lymphocytes and Bone Marrow for ADA− Immunodeficient Patients
Results indicate successful gene transfer into long-lasting progenitor cells, producing a functional multilineage progeny. Expand
Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs
It is reported that intra-arterial delivery of wild-type canine mesoangioblasts (vessel-associated stem cells) results in an extensive recovery of dystrophin expression, normal muscle morphology and function, and a remarkable clinical amelioration and preservation of active motility. Expand
Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study.
Infusion of TK-cells might be effective in accelerating immune reconstitution, while controlling GVHD and protecting patients from late mortality in those who are candidates for haploidentical stem-cell transplantation. Expand