Persistent low-level engraftment of rhesus peripheral blood progenitor cells transduced with the fanconi anemia C gene after conditioning with low-dose irradiation

Molecular Therapy

Volumn 3, Issue 6, 2001, Pages 911-919

  Kang, Elizabeth M ^a   Hanazano, Yutaka ^a   Frare, Pascale ^b   Vanin, Elio F ^b   De Witte, Moniek ^a   Metzger, Mark ^a   Liu, Johnson M ^a   Tisdale, John F ^a  

^a NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES   (United States)

^b ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

Autologous transplantation; Gene transduction; Hematopoietic stem cell; Nonmyeloablative conditioning

Indexed keywords

ANTIBIOTIC AGENT; BETA GALACTOSIDASE; CD34 ANTIGEN; FIBRONECTIN; FLT3 LIGAND; NEOMYCIN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; RECOMBINANT INTERLEUKIN 3; RECOMBINANT INTERLEUKIN 6; RECOMBINANT STEM CELL FACTOR; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; APHERESIS; ARTICLE; BIOLOGICAL THERAPY; BLOOD TRANSFUSION; CANCER RADIOTHERAPY; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; FANCONI ANEMIA; GENE SEQUENCE; GENE TARGETING; GENE THERAPY; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HOST; HUMAN; MACACA; NEUTROPENIA; NONHUMAN; PERIPHERAL BLOOD STEM CELL; PHENOTYPE; POLYMERASE CHAIN REACTION; POPULATION DYNAMICS; PREVENTION; RADIATION DOSE; RETROVIRUS; RHESUS MONKEY; RNA TRANSLATION;

ANIMALIA; MACACA; MACACA MULATTA; MURINAE; UNIDENTIFIED RETROVIRUS;

EID: 0034959561     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2001.0337     Document Type: Article

References (44)

1. Gene transfer for the eventual treatment of Fanconi's anemia
2. Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector
3. Gene marking and gene therapy directed at primary hematopoietic cells
4. Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor
5. Retrovirus-mediated gene transduction into canine peripheral blood repopulating cells
6. Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability
7. Introduction of a xenogeneic gene via hematopoietic stem cell leads to specific tolerance in a rhesus monkey model
8. + progenitors using clinically feasible methods
9. Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates
10. Induction by alkylating agents of sister chromatid exchanges and chromatid breaks in Fanconi's anemia
11. Long-term engraftment of normal and post-5-fluororacil murine marrow into normal nonmyeloablated mice
12. Enhanced engraftment of hematopoietic progenitor cells in mice treated with granulocyte colony-stimulating factor before low-dose irradiation: Implications for gene therapy
13. In vivo persistence of genetically-modified murine hematopoietic progenitors is not limited by the expression of the neo or GFP gene after full or minimal myeloablation
14. In vivo marking of rhesus monkey lymphocytes by adeno-associated viral vectors: Direct comparison with retroviral vectors
15. Green fluorescent protein retroviral vectors: Low titer and high recombination frequency suggest a selective disadvantage
16. Retroviral-mediated gene transfer into CD34-enriched human peripheral blood stem cells
17. Stem cell transplantation from HLA-matched related donor for Fanconi's anaemia: A retrospective review of the multicentric Italian experience on behalf of AIEOP-GITMO
18. Transplantation for Fanconi's anaemia: Long-term follow-up of fifty patients transplanted from a sibling donor after low-dose cyclophosphamide and thoraco-abdominal irradiation for conditioning
19. Transfer of the human glucocerebrosidase gene into hematopoietic stem cells of nonablated recipients: Successful engraftment and long-term expression of the transgene
20. Enhanced host defense after gene transfer in the murine p47phox-deficient model of chronic granulomatous disease
21. Lymphohematopoietic engraftment in minimally myeloablated hosts
22. Stem cell transplantation in the normal nonmyeloablated host: Relationship between cell dose, schedule, and engraftment
23. Repetitive bone marrow transplantation in nonmyeloablated recipients
24. + bone marrow cells
25. + hematopoietic cell transduction with cell-free retroviral vector preparations
26. Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone-marrow cells
27. + hematopoietic progenitor cells measured in vitro and in vivo
28. + cells in ADA-deficient SCID neonates
29. Functional correction of Fanconi anemia group A hematopoietic cells by retroviral gene transfer
30. Retroviral mediated gene transfer of the Fanconi anemia complementation group C gene to hematopoietic progenitors of group C patients
31. A functionally active retrovirus vector for gene therapy in Fanconi anemia group C
32. In vivo selection of wild-type hematopoietic stem cells in a murine model of Fanconi anemia
33. Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC)
34. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
35. Retroviral marking and transplantation of rhesus hematopoietic cells by nonmyeloablative conditioning
36. Efficient and durable gene marking of hematopoietic progenitor cells in nonhuman primates after nonablative conditioning
37. Immune response to green fluorescent protein: Implications for gene therapy
38. Preclinical protocol for in vivo selection of hematopoietic stem cells corrected by gene therapy in Fanconi anemia group C
39. Behavior of hematopoietic stem cells in a large animal
40. Engraftment of hematopoietic stem cells in nonmyeloablated and myeloablated hosts
41. Genetically-marked rhesus peripheral blood progenitor cells (PBPC) ex vivo expanded in the presence of fibronectin 296 contribute to both short and long term engraftment
42. Lymphohematopoietic stem cell engraftment