Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.

Autor: Carmo M; Infection, Immunity, Inflammation and Physiological Medicine Programme, Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK., Risma KA; 1] Division of Allergy/Immunology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [2] Division of Immunobiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [3] Division of Bone Marrow Transplant and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA., Arumugam P; 1] Division of Allergy/Immunology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [2] Division of Immunobiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [3] Division of Bone Marrow Transplant and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA., Tiwari S; 1] Division of Allergy/Immunology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [2] Division of Immunobiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [3] Division of Bone Marrow Transplant and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA., Hontz AE; 1] Division of Allergy/Immunology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [2] Division of Immunobiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [3] Division of Bone Marrow Transplant and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA., Montiel-Equihua CA; Infection, Immunity, Inflammation and Physiological Medicine Programme, Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK., Alonso-Ferrero ME; Infection, Immunity, Inflammation and Physiological Medicine Programme, Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK., Blundell MP; Infection, Immunity, Inflammation and Physiological Medicine Programme, Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK., Schambach A; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany., Baum C; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany., Malik P; 1] Division of Allergy/Immunology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [2] Division of Immunobiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [3] Division of Bone Marrow Transplant and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA., Thrasher AJ; Infection, Immunity, Inflammation and Physiological Medicine Programme, Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK., Jordan MB; 1] Division of Allergy/Immunology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [2] Division of Immunobiology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA [3] Division of Bone Marrow Transplant and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA., Gaspar HB; Infection, Immunity, Inflammation and Physiological Medicine Programme, Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2015 Apr; Vol. 23 (4), pp. 737-45. Date of Electronic Publication: 2014 Dec 19.

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.

Autor: Chiesa R; Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, UK., Gilmour K, Qasim W, Adams S, Worth AJ, Zhan H, Montiel-Equihua CA, Derniame S, Cale C, Rao K, Hiwarkar P, Hough R, Saudemont A, Fahrenkrog CS, Goulden N, Amrolia PJ, Veys P

Publikováno v: British journal of haematology [Br J Haematol] 2012 Mar; Vol. 156 (5), pp. 656-66. Date of Electronic Publication: 2012 Jan 09.

Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency.

Autor: Montiel-Equihua CA; Centre for immunodeficiency, Molecular immunology Unit, UCL institute of Child Health, London, UK., Thrasher AJ, Gaspar HB

Publikováno v: Stem cells and cloning : advances and applications [Stem Cells Cloning] 2009 Dec 22; Vol. 3, pp. 1-12. Date of Electronic Publication: 2009 Dec 22.