LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Science

Volumn 302, Issue 5644, 2003, Pages 415-419

  Hacein Bey Abina, S ^a   Von Kalle, C ^c,d   Schmidt, M ^c   McCormack, M P ^e   Wulffraat, N ^f   Leboulch, P ^g   Lim, A ^h   Osborne, C S ⁱ   Pawliuk, R ^g   Morillon, E ^a   Sorensen, R ^o   Forster, A ^e   Fraser, P ⁱ   Cohen, J I ^k   De Saint Basile, G ^a   Alexander, I ^l   Wintergerst, U ^m   Frebourg, T ⁿ   Aurias, A ^o   Stoppa Lyonnet, D ^o   Romana, S ^a   Radford Weiss, I ^a   Gross, F ^a   Valensi, F ^b   Delabesse, E ^b   Macintyre, E ^b   Sigaux, F ^o   Soulier, J ⁿ   Leiva, L E ^j   Wissler, M ^c   Prinz, C ^c   Rabbitts, T H ^e   Le Deist, F ^a   Fischer, A ^a   Cavazzana Calvo, M ^a   more..

^a HÔPITAL NECKER ENFANTS MALADES   (France)

^b CNRS   (France)

^c UNIVERSITY OF FREIBURG   (Germany)

^d CINCINNATI CHILDREN S HOSPITAL RESEARCH FOUNDATION   (United States)

^e MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

^f UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^g BRIGHAM AND WOMEN S HOSPITAL   (United States)

^h INSTITUT PASTEUR   (France)

ⁱ BABRAHAM INSTITUTE   (United Kingdom)

^j LOUISIANA STATE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^k NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^l CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^m LUDWIG MAXIMILIANS UNIVERSITY   (Germany)

ⁿ INSERM   (France)

^o INSTITUT CURIE   (France)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CELLS; CLONING; GENE TRANSFER; PATIENT TREATMENT; VIRUSES;

GENE PROMOTER;

IMMUNOLOGY;

CYTOKINE RECEPTOR; GAMMA CYTOKINE RECEPTOR SUBUNIT; RECEPTOR SUBUNIT; RETROVIRUS VECTOR; UNCLASSIFIED DRUG; DNA BINDING PROTEIN; INTERLEUKIN 2 RECEPTOR; LMO2 PROTEIN, HUMAN; METALLOPROTEIN;

ANEMIA; ARTICLE; AUTOLOGOUS BONE MARROW TRANSPLANTATION; BONE MARROW CELL; CASE REPORT; CELL PROLIFERATION; COMBINED IMMUNODEFICIENCY; CONTROLLED STUDY; GAMMA CHAIN DEFICIENCY; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; LMO2 GENE PROMOTER; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTO ONCOGENE; SPLENOMEGALY; T LYMPHOCYTE; VIRUS DNA CELL DNA INTERACTION; X CHROMOSOME LINKAGE; CELL CLONE; CLINICAL TRIAL; GENE EXPRESSION REGULATION; GENE VECTOR; GENETIC TRANSCRIPTION; GENETICS; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; INFANT; PHYSIOLOGY; PROMOTER REGION; RETROVIRUS; T CELL LEUKEMIA; VIRUS REPLICATION;

INSERTION SEQUENCES; UNIDENTIFIED RETROVIRUS;

CLINICAL TRIALS; CLONE CELLS; DNA-BINDING PROTEINS; GENE EXPRESSION REGULATION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; INFANT; LEUKEMIA, T-CELL, ACUTE; METALLOPROTEINS; MUTAGENESIS, INSERTIONAL; PROMOTER REGIONS (GENETICS); PROTO-ONCOGENES; RECEPTORS, INTERLEUKIN-2; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY; T-LYMPHOCYTES; TRANSCRIPTION, GENETIC; VIRUS INTEGRATION; VIRUS REPLICATION;

EID: 0142084745     PISSN: 00368075     EISSN: None     Source Type: Journal    
DOI: 10.1126/science.1088547     Document Type: Article

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41. We are indebted to the families of the patients for their continuous support; to the medical and nursing staff of the Unité d'Immunologie et d'Hématologie Pédiatriques, Hôpital des Enfants Malades, for patient care; to F. Calvo, A. O. Cavazzana, D. Papadopoulo, P. Kourilsky, H. Bruzzoni-Giovanelli, C. Thibaut, P. Kastner, M. Bonneville, H. Vié, E. Vivier, and P. Paule for their contribution to the study; and to C. Hue for technical help. Supported by grants from INSERM; the Bundesministerium für Bildung, Wissenschaft, Forschung und Technologie; Deutsche Forschung Gemeinschaft; the Association Française contre les Myopathies; the Programme Hospitalier de Recherche CLinique of the Health Ministry (France); Assistance Publique-Hôpitaux de Paris; European Community contract no. QLK3-CT 2001 (G. Wagemaker, coordinator); and the Jeffrey Modell Foundation.