Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

Nature Medicine

Volumn 8, Issue 5, 2002, Pages 423-425

  Aiuti, Alessandro ^a   Vai, Sergio ^a   Mortellaro, Alessandra ^a   Casorati, Giulia ^b   Ficara, Francesca ^a   Andolfi, Grazia ^a   Ferrari, Giuliana ^a   Tabucchi, Antonella ^c   Carlucci, Filippo ^c   Ochs, Hans D ^d   Notarangelo, Luigi D ^e   Roncarolo, Maria Grazia ^a   Bordignon, Claudio ^a,f  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b SAN RAFFAELE HOSPITAL   (Italy)

^c UNIVERSITY OF SIENA   (Italy)

^d UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^e Clinica Pediatrica   (Italy)

^f VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; MACROGOL;

ADENOSINE DEAMINASE DEFICIENCY; CASE REPORT; CLINICAL FEATURE; COMBINED IMMUNODEFICIENCY; ENZYME DEFICIENCY; ENZYME REPLACEMENT; GENE THERAPY; HUMAN; IMMUNE RESPONSE; LETTER; PERIPHERAL LYMPHOCYTE; PRIORITY JOURNAL; TREATMENT OUTCOME;

ADENOSINE DEAMINASE; ANIMALS; CATTLE; GENE THERAPY; HUMANS; LYMPHOCYTE TRANSFUSION; SEVERE COMBINED IMMUNODEFICIENCY; T-LYMPHOCYTES;

EID: 18444376150     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm0502-423     Document Type: Letter

References (11)

1. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients
2. T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
3. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency
4. Bone marrow gene transfer in three patients with adenosine deaminase deficiency
5. Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency
6. Antibody responses to bacteriophage phi X 174 in patients with adenosine deaminase deficiency
7. Severe combined immunodeficiency and adenosine deaminase deficiency
8. Bone marrow transplantation only partially restores purine metabolites to normal in adenosine deaminase-deficient patients
9. + cells in ADA-deficient SCID neonates
10. Adenosine deaminase deficiency: Genotype-phenotype correlations based on expressed activity of 29 mutant alleles
11. Transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA-deficient SCID