Adenosine deaminase gene therapy protocol revisited

Molecular Therapy

Volumn 5, Issue 2, 2002, Pages 96-97

  Kohn, Donald B ^a  

^a CHILDREN S HOSPITAL LOS ANGELES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; BUSULFAN; CD34 ANTIGEN; CYCLOPHOSPHAMIDE; FIBRONECTIN; FLT3 LIGAND; INTERLEUKIN 3; RETROVIRUS VECTOR; STEM CELL FACTOR; THROMBOPOIETIN;

ARTICLE; BONE MARROW CELL; BONE MARROW TRANSPLANTATION; CLINICAL PROTOCOL; COMBINED IMMUNODEFICIENCY; CONTROLLED STUDY; DOSE RESPONSE; ENZYME REPLACEMENT; GENE THERAPY; HUMAN; NATURAL KILLER CELL; NEUTROPENIA; OPPORTUNISTIC INFECTION; T LYMPHOCYTE; VIRAL GENE DELIVERY SYSTEM; WHOLE BODY RADIATION;

UNIDENTIFIED RETROVIRUS;

EID: 0036169489     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2002.0535     Document Type: Note

References (6)

1. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
2. T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
3. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates
4. Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency
5. T-cell lines from 2 patients with adenosine deaminase (ADA) deficiency showed the restoration of ADA activity resulted from the reversion of an inherited mutation
6. Correction of ADA-SCID defect without PEG-ADA therapy by stem/progenitor cell gene therapy combined with a non-myeloablative conditioning