Gene therapy for adenosine deaminase deficiency

Current Opinion in Allergy and Clinical Immunology

Volumn 3, Issue 6, 2003, Pages 461-466

  Aiuti, Alessandro ^a   Ficara, Francesca ^a   Cattaneo, Federica ^a   Bordignon, Claudio ^a   Roncarolo, Maria Grazia ^a  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

Author keywords

Clinical trial; Gene transfer in hematopoietic stem cells; Gene transfer in peripheral blood lymphocytes; Severe combined immunodeficiency

Indexed keywords

ADENOSINE DEAMINASE; BUSULFAN; CD34 ANTIGEN; MACROGOL;

ANTIBODY RESPONSE; COMBINED IMMUNODEFICIENCY; DRUG SAFETY; ENZYME DEFICIENCY; GENE THERAPY; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; PERIPHERAL LYMPHOCYTE; PRIORITY JOURNAL; REVIEW; T LYMPHOCYTE; TRANSGENE; CLINICAL TRIAL; GENETICS; LYMPHOCYTE; METABOLISM; METHODOLOGY;

ADENOSINE DEAMINASE; CLINICAL TRIALS; GENE THERAPY; HEMATOPOIETIC STEM CELLS; HUMANS; LYMPHOCYTES; SEVERE COMBINED IMMUNODEFICIENCY;

EID: 2642567436     PISSN: 15284050     EISSN: None     Source Type: Journal    
DOI: 10.1097/00130832-200312000-00007     Document Type: Review

References (28)

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16. Muul LM, Tuschong LM, Soenen SL, et al. Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. Blood 2003; 101:2563-2569. An interesting study on the long-term follow up of the first two ADA-SCID patients treated with PBL gene therapy.
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18. + cell gene therapy. The data show that few progenitor engrafted and a single progenitor gave rise to the majority of mature corrected lymphocytes.
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