Expression of human adenosine deaminase in mice reconstituted with retrovirus-transduced hematopoietic stem cells
- Massachusetts Institute of Technology, Cambridge (USA)
Recombinant retroviruses encoding human adenosine deaminase have been used to infect murine hematopoietic stem cells. In bone marrow transplant recipients reconstituted with the genetically modified cells, human ADA was detected in peripheral blood mononuclear cells of the recipients for at least 6 months after transplantation. In animals analyzed in detail 4 months after transplantation, human ADA and proviral sequences were detected in all hematopoietic lineages; in several cases, human ADA activity exceeded the endogenous activity. These studies demonstrate the feasibility of introducing a functional human ADA gene into hematopoietic stem cells and obtaining expression in multiple hematopoietic lineages long after transplantation. This approach should be helpful in designing effective gene therapies for severe combined immunodeficiency syndromes in humans.
- OSTI ID:
- 6941240
- Journal Information:
- Proceedings of the National Academy of Sciences of the United States of America; (USA), Vol. 87:1; ISSN 0027-8424
- Country of Publication:
- United States
- Language:
- English
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BONE MARROW CELLS
GENE RECOMBINATION
NON-PEPTIDE C-N HYDROLASES
GENE REGULATION
DNA HYBRIDIZATION
ELECTROPHORESIS
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ONCOGENIC VIRUSES
PHOSPHORUS 32
STEM CELLS
ANIMAL CELLS
ANIMALS
BETA DECAY RADIOISOTOPES
BETA-MINUS DECAY RADIOISOTOPES
CONNECTIVE TISSUE CELLS
DAYS LIVING RADIOISOTOPES
ENZYMES
HYBRIDIZATION
HYDROLASES
ISOTOPES
LIGHT NUCLEI
MAMMALS
MICROORGANISMS
NUCLEI
ODD-ODD NUCLEI
PARASITES
PHOSPHORUS ISOTOPES
RADIOISOTOPES
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SOMATIC CELLS
VERTEBRATES
VIRUSES
550201* - Biochemistry- Tracer Techniques