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Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells
Universal gene-edited CAR19 T cells eliminate infant leukemia. CAR sharing Chimeric antigen receptor (CAR) T cells can be very effective in treating acute lymphocytic leukemia. Unfortunately, theseExpand
Omission of in vivo T‐cell depletion promotes rapid expansion of naïve CD4+ cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant
Umbilical cord blood transplant (UCBT) is associated with impaired early immune reconstitution. This might be explained by a lower T‐cell dose infused, the naivety of cord blood T‐cells and the useExpand
Human Coronavirus OC43 Associated with Fatal Encephalitis.
In this case report, severe encephalitis with no established cause developed in a child with SCID. Through deep sequencing, human coronavirus OC43 was identified in brain tissue.
FOXRED1, encoding an FAD-dependent oxidoreductase complex-I-specific molecular chaperone, is mutated in infantile-onset mitochondrial encephalopathy.
Clinical and Molecular Genetics Unit, UCL Institute of Child Health, London WC1N 1EH, UK, Dino Ferrari Centre, Department of Neurological Sciences, University of Milan, I.R.C.C.S. Foundation Ca’Expand
Immunotherapy of HCC metastases with autologous T cell receptor redirected T cells, targeting HBsAg in a liver transplant patient.
HBV-DNA integration frequently occurs in HBV-related hepatocellular carcinoma (HCC), but whether HBV antigens are expressed in HCC cells and can be targeted by immune therapeutic strategies remainsExpand
Astrovirus VA1/HMO-C: An Increasingly Recognized Neurotropic Pathogen in Immunocompromised Patients
Brain biopsy from a child with unknown cause of encephalopathy was deep-sequenced. Astrovirus VA1/HMO-C was identified, highly divergent from human astroviruses and 95% identical to astrovirusExpand
How I treat severe combined immunodeficiency.
Severe combined immunodeficiency (SCID) arises from different genetic defects associated with lymphocyte development and function and presents with severe infections. Allogeneic hematopoietic stemExpand
Clinical and immunological manifestations of patients with atypical severe combined immunodeficiency.
Hypomorphic mutations in genes associated with severe combined immunodeficiency (SCID) or Omenn syndrome can also cause milder immunodeficiencies. We report 10 new patients with such "atypical" SCIDExpand
Cord blood T cells mediate enhanced antitumor effects compared with adult peripheral blood T cells.
Unrelated cord blood transplantation (CBT) without in vivo T-cell depletion is increasingly used to treat high-risk hematologic malignancies. Following T-replete CBT, naïve CB T cells undergo rapidExpand
Lentiviral vectors for T-cell suicide gene therapy: preservation of T-cell effector function after cytokine-mediated transduction.
Retroviral transfer of the Herpes Simplex thymidine kinase (HSVTK) suicide gene to donor T cells has been used as a safety strategy against graft-versus-host disease following allogeneic stem cellExpand