Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

Nature Medicine

Volumn 9, Issue 4, 2003, Pages 463-468

  Schmidt, Manfred ^a   Carbonaro, Denise A ^b   Speckmann, Carsten ^a   Wissler, Manuela ^a   Bohnsack, John ^c   Elders, Melissa ^d   Aronow, Bruce J ^e   Nolta, Jan A ^e   Kohn, Donald B ^b   Von Kalle, Christof ^a,f  

^a UNIVERSITY OF FREIBURG   (Germany)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^c UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^f WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; CD34 ANTIGEN; RETROVIRUS VECTOR; T LYMPHOCYTE RECEPTOR;

AMINO ACID SEQUENCE; ARTICLE; BONE MARROW CELL; CELL ISOLATION; CLINICAL TRIAL; CLONAL VARIATION; COMBINED IMMUNODEFICIENCY; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; ENZYME DEFICIENCY; GENE AMPLIFICATION; GENE REARRANGEMENT; GENE TRANSFER; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; LEUKEMIA; LYMPHOCYTE CLONE; MALIGNANT TRANSFORMATION; MARKER GENE; NEWBORN; NUCLEOTIDE SEQUENCE; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; STEM CELL; T LYMPHOCYTE; THYMUS; UMBILICAL CORD BLOOD; VIRAL GENE THERAPY;

ADENOSINE DEAMINASE; ANTIGENS, CD34; CLONE CELLS; FETAL BLOOD; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; INFANT, NEWBORN; POLYMERASE CHAIN REACTION; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY; T-LYMPHOCYTES; TRANSDUCTION, GENETIC;

ADA; UNIDENTIFIED RETROVIRUS;

EID: 0345144017     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm844     Document Type: Article

References (16)

1. Kohn, D.B. et al. Engraftment of gene-modified cells from umbilical cord blood in neonates with adenosine deaminase deficiency. Nat. Med. 1, 1017-1026 (1995).
2. - cells in ADA-deficient SCID neonates. Nat. Med. 4, 775-780 (1998).
3. Schmidt, M. et al. Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples. Hum. Gene Ther. 12, 743-749 (2001).
4. Schmidt, M. et al. Polyclonal long-term repopulating stem cell clones in a primate model. Blood 100, 2737-2743 (2002).
5. Hacein-Bey-Abina, S. et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348, 255-256 (2003).
6. Marshall, E. Gene therapy. Second child in French trial is found to have leukemia. Science 299, 320 (2003).
7. Capel, B., Hawley, R.G. & Mintz, B. Long- and short-lived murine hematopoietic stem cell clones individually identified with retroviral integration markers. Blood 75, 2267-2270 (1990).
8. Morrison, S.I. & Weissman, I.L. The long-term repopulating subset of hematopoietic stem cells is deterministic and isolatable by phenotype. Immunity 1, 661-673 (1994).
9. Glimm, H. et al. Previously undetected human hematopoietic cell populations with short-term repopulating activity selectively engraft NOD/SCID-β2 microglobulinnull mice. J. Clin. Invest. 107, 199-206 (2001).
10. Willenbrock, K. et al. Analysis of T-cell subpopulations in T-cell non-Hodgkin's lymphoma of angioimmunoblastic lymphadenopathy with dysproteinemia type by single target gene amplification of T cell receptor-β gene rearrangements. Am. J. Pathol. 158, 1851-1857 (2001).
11. Tisdale, I.F. et al. Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability. Blood 92, 1131-1141 (1998).
12. Kim, H.I. et al. Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates. Blood 96, 1-8 (2000).
13. Jensen, M.C. et al. Human T lymphocyte genetic modification with naked DNA. Mol. Ther. 1, 49-55 (2000).
14. Kent, W.I. BLAT: the BLAST-like Alignment Tool. Genome Res. 12, 656-664 (2002).
15. Kent, W.I. et al. The Human Genome Browser at UC5C. Genome Res. 12, 996-1006 (2002).
16. Lander, E. et al. Initial sequencing and analysis of the human genome. Nature 409, 860-892 (2001).