Affiliation: University of Southern California
- Factors affecting thymic function after allogeneic hematopoietic stem cell transplantationK Weinberg
Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital Los Angeles, CA 90027, USA
Blood 97:1458-66. 2001..Collectively, these data suggest that thymopoiesis is inhibited by GVHD. Larger studies will be needed to determine the independent contributions of age and preparative regimen to post-transplant thymopoietic capacity...
- Study of thymic size and function in children and adolescents with treatment refractory systemic sclerosis eligible for immunoablative therapyA Reiff
Divisions of Rheumatology, Radiology and Bone Marrow Transplantation, Department of Pediatrics, University of Southern California, Keck School of Medicine, Children s Hospital Los Angeles, CA 90027, USA
Clin Immunol 133:295-302. 2009..A thorough evaluation of immune and thymic functions to identify those patients prior to HSCT is recommended...
- Exclusion of candidate genetic loci for Duane retraction syndromeS Ott
Division of Ophthalmology, Children s Hospital Los Angeles, University of Southern California School of Medicine, 90064, USA
Am J Ophthalmol 127:358-60. 1999..To report preliminary linkage analysis of a large Hispanic family showing autosomal dominant inheritance for Duane retraction syndrome...
- Successful hematopoietic stem cell transplantation for Niemann-Pick disease type BAmi J Shah
Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital Los Angeles, CA 90027, USA
Pediatrics 116:1022-5. 2005..Five years after her second HSCT, her sphingomyelinase levels are within normal levels, she has no pulmonary symptoms, and aside from persistent graft versus host disease, she is doing well...
- Successful immune reconstitution decreases leukemic relapse and improves survival in recipients of unrelated cord blood transplantationRobertson Parkman
Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital Los Angeles, The Saban Research Institute, 4650 Sunset Boulevard, Mail Stop 62, Los Angeles, CA 90027, USA
Biol Blood Marrow Transplant 12:919-27. 2006..Successful antigen-specific immune reconstitution after unrelated cord blood transplantation results in decreased leukemic relapse and improved overall survival...
- [Treatment progress of Duchenne Muscular Dystrophy (DMD)]Elzbieta Monika Smogorzewska
Hematopoietic Stem Cell Processing Laboratory, Research Immunology, Children Hospital LA, Los Angeles, USA
Med Wieku Rozwoj 8:25-32. 2004..In the future it may be possible for mesenchymal cells to be isolated, ex vivo expanded and transplanted into patients with muscle diseases...
- [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]Elzbieta Monika Smogorzewska
Research Immunology, Children Hospital LA, Los Angeles, CA 90027, USA
Med Wieku Rozwoj 7:27-34. 2003..This case is being investigated to determine whether the genetic manipulations of the patient's HSC could be the reason for mutagenesis and how other factors could have contributed to this unfortunate event...
- Preponderant mitotic activity of nonleukemic cells plays an important role in failures to detect abnormal clone in childhood acute lymphoblastic leukemiaShi Qi Wu
Department of Pediatrics, Children s Hospital Los Angeles, Keck School of Medicine, University of Southern California, 4650 Sunset Blvd, Mailstop 11, Los Angeles, CA 90027, U S A
J Pediatr Hematol Oncol 25:520-5. 2003..Therefore, karyotypically "normal" childhood ALL patients should undergo FISH studies to rule out the presence of t(12;21) and/or hyperdiploid clone...
- Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignanciesAmi J Shah
Department of Pediatrics, Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital, Los Angeles, CA 90027, USA
J Pediatr Hematol Oncol 30:411-8. 2008..Although some patients have acute deficits that appear and improve over time, other patients have progressive declines in neurocognitive function that are chronic...
- Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-upAmi J Shah
Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital, Los Angeles, CA 90027, USA
Biol Blood Marrow Transplant 8:221-8. 2002..Although toxicity is considerable following a second transplantation, the major causes of mortality continue to be relapse and infection...
- Pre- and post-natal treatment of hemophagocytic lymphohistiocytosisAmi J Shah
Division of Research Immunology, Childrens Hospital Los Angeles, California 90027, USA
Pediatr Blood Cancer 52:139-42. 2009..We report on the first patient to receive post-natal HSCT for HLH after receiving in utero chemotherapy for disease stabilization...
- Human CD34(+) and CD34(+)CD38(-) hematopoietic progenitors in sickle cell disease differ phenotypically and functionally from normal and suggest distinct subpopulations that generate F cellsLori Luck
Division of Hematology Oncology, Childrens Hospital Los Angeles, Los Angeles, CA 90027, USA
Exp Hematol 32:483-93. 2004..Sickle cell disease (SCD) is remarkable for stress erythropoiesis. We investigated the progenitor populations contributing to erythroid stress...
- Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimenNoboru Mitsuhashi
Childrens Hospital of Los Angeles, Department of Cardiothoracic Surgery, Los Angeles, CA 90027, USA
Blood 107:2286-93. 2006..Mice reconstituted with GalT accepted alphaGal+ heart grafts over 100 days. Transduction with lentiviral vectors results in chimerism at levels sufficient to induce long-term tolerance under nonmyeloablative conditions...
- Localization of a gene for Duane retraction syndrome to chromosome 2q31B Appukuttan
Division of Ophthalmology, Childrens Hospital Los Angeles, Los Angeles, CA, 90027, USA
Am J Hum Genet 65:1639-46. 1999..Coding sequences for these genes were normal or had genetic alterations unlikely to be responsible for the DRS phenotype. Identifying the gene responsible for DRS may lead to an improved understanding of early cranial-nerve development...
- Protection from thymic epithelial cell injury by keratinocyte growth factor: a new approach to improve thymic and peripheral T-cell reconstitution after bone marrow transplantationDullei Min
Division of Research Immunology BMT, Childrens Hospital Los Angeles, CA 90027, USA
Blood 99:4592-600. 2002..Furthermore, thymic injury and prolonged posttransplantation immune deficiency in BMT recipients can be prevented by KGF administration...
- Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case reportBarbara C Engel
Division of Research Immunology Bone Marrow Transplantation, General Clinical Research Center, Department of Pathology, Children s Hospital Los Angeles, CA 90027, and Baylor College of Medicine, Houston, TX, USA
Blood 109:503-6. 2007..The confounding effects of this preexisting marrow cytogenetic abnormality on the response to gene transfer highlights another challenge of gene therapy with the use of autologous hematopoietic stem cells...
- Gene therapy for adenosine deaminase deficiencyR Parkman
Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital Los Angeles, California, USA
Annu Rev Med 51:33-47. 2000....
- Severe combined immunodeficiency due to a specific defect in the production of interleukin-2K Weinberg
Division of Research Immunology, Childrens Hospital Los Angeles, CA 90027
N Engl J Med 322:1718-23. 1990
- Identification, functional analysis and expression in a heterotopic heart transplant model of CXCL9 in the ratNoboru Mitsuhashi
Division of Cardiothoracic Surgery, Department of Cardiothoracic Surgery, Children s Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, CA 90027, USA
Immunology 112:87-93. 2004..These findings indicate that rat CXCL9 has an in vivo role in the infiltration of CD4+ T cells in the transplanted graft...
- Mouse mesenchymal stem cells expressing PAX-FKHR form alveolar rhabdomyosarcomas by cooperating with secondary mutationsYue Xin Ren
Department of Pathology and Laboratory Medicine, Children s Hospital Los Angeles, CA 90027, USA
Cancer Res 68:6587-97. 2008..Our results show the critical role played by PAX-FKHR in determining the molecular, myogenic, and histologic phenotype of ARMS. More importantly, we identify MSCs as a progenitor that can give rise to ARMS...
- Severe papillomavirus infection progressing to metastatic squamous cell carcinoma in bone marrow-transplanted X-linked SCID dogsMichael H Goldschmidt
Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, 3900 Delancey St, Philadelphia, PA 19104, USA
J Virol 80:6621-8. 2006..Canine XSCID therefore provides an ideal animal model with which to study the role of the gammac-dependent signaling pathway in the response to papillomavirus infections and the progression of these viral infections to metastatic SCC...
- Transplantation of X-linked severe combined immunodeficient dogs with CD34+ bone marrow cellsBrian J Hartnett
Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia 19104, USA
Biol Blood Marrow Transplant 8:188-97. 2002....
- Importance of interleukin-7 in the development of experimental graft-versus-host diseaseBrile Chung
Division of Stem Cell Transplantation, Department of Pediatrics, Stanford University School of Medicine, Stanford, California, USA
Biol Blood Marrow Transplant 14:16-27. 2008..These experiments demonstrate that IL-7 is an important factor in the development of GVHD, presumably by supporting the survival, proliferation, and possibly activation of alloreactive donor-derived T cells in the recipients...
- T cell repertoire development in XSCID dogs following nonconditioned allogeneic bone marrow transplantationWilliam Vernau
Department of Veterinary Pathology, Microbiology and Immunology, School of Veterinary Medicine, University of California, Davis, California, USA
Biol Blood Marrow Transplant 13:1005-15. 2007..However, in contrast to transplanted XSCID boys who show a significant decline in their T cell diversity by 10 to 12 years following BMT, transplanted XSCID dogs maintain a polyclonal, diverse T cell repertoire through midlife...
- Prevention of graft-versus-host disease by anti IL-7Ralpha antibodyBrile Chung
Division of Stem Cell Transplantation, Department of Pediatrics, Stanford University School of Medicine, Palo Alto, CA 94304, USA
Blood 110:2803-10. 2007..Blockade of IL-7R by anti-IL-7Ralpha antibody resulted in elimination of alloreactive T cells, prevention of GVHD, and improvement of donor T-cell reconstitution...
- The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplantsAmi J Shah
Department of Pediatrics, Keck School of Medicine, Los Angeles, California, USA
Biol Blood Marrow Transplant 13:584-93. 2007..Further studies are warranted to assess if a lack of difference in infection rates are still demonstrated in larger cohorts...
- Retinoid-induced G1 arrest and differentiation activation are associated with a switch to cyclin-dependent kinase-activating kinase hypophosphorylation of retinoic acid receptor alphaJiwei Wang
Department of Pathology, Division of Research Immunology Bone Marrow Transplant, Childrens Hospital Los Angeles Research Institute, Los Angeles, California 90027, USA
J Biol Chem 277:43369-76. 2002....
- Antigen-specific T-lymphocyte function after cord blood transplantationGeoff Cohen
The EMMES Corporation, Rockville, Maryland, USA
Biol Blood Marrow Transplant 12:1335-42. 2006..These results indicate that naive T lymphocytes present in the HSC inoculum can contribute to the generation of antigen-specific T-lymphocyte immunity early after transplantation...
- Early use of drastic therapyKenneth I Weinberg
N Engl J Med 352:2124-6. 2005