Treatment of patients with severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency by autologous transplantation of genetically modified bone marrow cells.

Human Gene TherapyVol. 3, No. 5 News and CommentsTreatment of Patients with Severe Combined Immunodeficiency due to Adenosine Deaminase (ADA) Deficiency by Autologous Transplantation of Genetically Modified Bone Marrow Cells. University Hospital Leiden and Institute of Applied Radiobiology and Immunology TNO, The NetherlandsINVESTIGATORS P. M. Hoogerbrugge, J. M. J. J. Vossen, V. W. v. Beusechem, and D. ValerioINVESTIGATORS P. M. HoogerbruggeSearch for more papers by this author, J. M. J. J. VossenSearch for more papers by this author, V. W. v. BeusechemSearch for more papers by this author, and D. ValerioSearch for more papers by this authorPublished Online:13 Jun 2008https://doi.org/10.1089/hum.1992.3.5-553AboutSectionsPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail FiguresReferencesRelatedDetailsCited byTargeted genome editing for the correction or alleviation of primary ImmunodeficienciesOutcomes in Two Japanese Adenosine Deaminase-Deficiency Patients Treated by Stem Cell Gene Therapy with No Cytoreductive Conditioning15 April 2015 | Journal of Clinical Immunology, Vol. 35, No. 4Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plansBlood, Vol. 120, No. 18Retroviral transduction efficiency of G-CSF+SCF–mobilized peripheral blood CD34+ cells is superior to G-CSF or G-CSF+Flt3-L–mobilized cells in nonhuman primatesBlood, Vol. 101, No. 6What can cardiovascular gene transfer learn from genomics: and vice versa?Physiological Genomics, Vol. 11, No. 3Genetically modified bone marrow continuously supplies anti-inflammatory cells and suppresses renal injury in mouse Goodpasture syndromeBlood, Vol. 98, No. 1Protection of Hematopoietic Stem Cells from Chemotherapy-Induced Toxicity by Multidrug-Resistance 1 Gene TransferGene therapy of primary immunodeficienciesSpringer Seminars in Immunopathology, Vol. 19, No. 4Interactions between the Immune System and Gene Therapy Vectors: Bidirectional Regulation of Response and Expression**Received for publication September 19, 1997Genetic engineering: Moral aspects and control of practiceJournal of Assisted Reproduction and Genetics, Vol. 14, No. 6Cytokines and prostaglandins in immune homeostasis and tissue destruction in periodontal diseasePeriodontology 2000, Vol. 14, No. 1Retroviral gene therapy in hematopoietic diseasesJournal of Clinical Apheresis, Vol. 12, No. 4Cell-Based Myocardial Protein DeliveryChapter 18 The advantages of liposome-based gene therapy: A comparison of viral versus liposome-based gene deliveryTHE USE OF GENE THERAPY FOR IMMUNODEFICIENCY DISEASERadiologic Clinics of North America, Vol. 16, No. 3THE USE OF GENE THERAPY FOR IMMUNODEFICIENCY DISEASEImmunology and Allergy Clinics of North America, Vol. 16, No. 3Bone Marrow and Clinical Gene Therapy GABRIELE D. SCHMIDT-WOLF and INGO G.H. SCHMIDT-WOLF27 March 2009 | Journal of Hematotherapy, Vol. 4, No. 6Recent advances in the application of gene therapy to human diseaseThe American Journal of Medicine, Vol. 99, No. 5Porphyrie érythropoïétique congénitale. À propos d'un cas fatal en période néonatale dûà une hémolyse aiguë avec insuffisance hépatiqueArchives de Pédiatrie, Vol. 2, No. 8Porphyrias: Animal models and prospects for cellular and gene therapyJournal of Bioenergetics and Biomembranes, Vol. 27, No. 2The contribution of marker gene studies to hemopoietic stem cell therapies23 December 2008 | STEM CELLS, Vol. 13, No. 5Retroviral transfer of the multidrug resistance ‐1 gene into lineage‐committed and primitive hemopoietic cellsStem Cells, Vol. 13, No. S3Is there a Role for Transfusion Medicine in the Genetic Correction of Genetic Disorders and other Diseases?Autologous Bone Marrow Transplantation27 September 2008 | Critical Reviews in Clinical Laboratory Sciences, Vol. 32, No. 1An Evaluation of Receptor-Mediated Gene Transfer Using Synthetic DNA-Ligand ComplexesEuropean Journal of Biochemistry, Vol. 226, No. 2An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexesCurrent trends and future directions in the genetic therapy of human neoplastic diseaseCancer, Vol. 72, No. 7Gene Transfer into Human Hematopoietic Progenitor Cells: A Review of Current Clinical Protocols MALCOLM K. BRENNER8 May 2009 | Journal of Hematotherapy, Vol. 2, No. 1 Volume 3Issue 5Oct 1992 To cite this article:INVESTIGATORS P. M. Hoogerbrugge, J. M. J. J. Vossen, V. W. v. Beusechem, and D. Valerio.Treatment of Patients with Severe Combined Immunodeficiency due to Adenosine Deaminase (ADA) Deficiency by Autologous Transplantation of Genetically Modified Bone Marrow Cells. University Hospital Leiden and Institute of Applied Radiobiology and Immunology TNO, The Netherlands.Human Gene Therapy.Oct 1992.553-558.http://doi.org/10.1089/hum.1992.3.5-553Published in Volume: 3 Issue 5: June 13, 2008PDF download