A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency [1]

New England Journal of Medicine

Volumn 348, Issue 3, 2003, Pages 255-256

  Hacein Bey Abina, Salima ^a   Von Kalle, Christof ^d   Schmidt, Manfred ^b   Le Deist, Françoise ^a   Wulffraat, Nicolas ^c   McIntyre, Elisabeth ^a   Radford, Isabelle ^a   Villeval, Jean Luc ^d   Fraser, Christopher C ^d   Cavazzana Calvo, Marina ^a   Fischer, Alain ^a  

^a HÔPITAL NECKER ENFANTS MALADES   (France)

^b UNIVERSITY OF FREIBURG   (Germany)

^c UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^d NONE

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN;

ACUTE LYMPHOBLASTIC LEUKEMIA; ACUTE LYMPHOCYTIC LEUKEMIA; CHROMOSOME 11; CHROMOSOME TRANSLOCATION; COMBINED IMMUNODEFICIENCY; GENE INSERTION; GENE LOCUS; GENE MUTATION; GENE THERAPY; GENE TRANSFER; GENETIC PREDISPOSITION; HEPATOSPLENOMEGALY; HUMAN; LETTER; LYMPHOCYTE COUNT; LYMPHOCYTOSIS; LYMPHOPROLIFERATIVE DISEASE; MEDULLOBLASTOMA; OUTCOMES RESEARCH; PRIORITY JOURNAL; RETROVIRUS; STEM CELL TRANSPLANTATION; VIRAL GENE DELIVERY SYSTEM; X CHROMOSOME LINKAGE;

GENETICS AND REPRODUCTION;

CHROMOSOMES, HUMAN, PAIR 11; CHROMOSOMES, HUMAN, X; DNA-BINDING PROTEINS; GENE EXPRESSION; GENE THERAPY; HUMANS; LEUKEMIA, LYMPHOCYTIC, ACUTE; LINKAGE (GENETICS); LYMPHOCYTE COUNT; METALLOPROTEINS; MUTAGENESIS, INSERTIONAL; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY;

EID: 0037448352     PISSN: 00284793     EISSN: None     Source Type: Journal    
DOI: 10.1056/NEJM200301163480314     Document Type: Letter

References (5)

1. Hacein-Bey-Abina S, Le Deist F, Carlier F, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002;346:1185-93.
2. Schmidt M, Zickler P, Hoffmann G, et al. Polyclonal long-term repopulating stem cell clones in a primate model. Blood 2002;100:2737-43.
3. Rabbitts TH, Bucher K, Chung G, Grutz G, Warren A, Yamada Y. The effect of chromosomal translocations in acute leukemias: the LMO2 paradigm in transcription and development. Cancer Res 1999;59:Suppl:1794s-1798s.
4. Stocking C, Bergholz U, Friel J, et al. Distinct classes of factor-independent mutants can be isolated after retroviral mutagenesis of a human myeloid stem cell line. Growth Factors 1993;8:197-209.
5. Buckley RH, Schiff SE, Schiff RI, etal. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999;340:508-16.