Expression of human adenosine deaminase in mice reconstituted with retrovirus-transduced hematopoietic stem cells

Journal Article · Sun Dec 31 23:00:00 EST 1989 · Proceedings of the National Academy of Sciences of the United States of America; (USA)

DOI:https://doi.org/10.1073/pnas.87.1.439· OSTI ID:6941240

Wilson, J M; Danos, O; Grossman, M; Raulet, D H; Mulligan, R C ^[1]

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1. 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), Journal Name: Proceedings of the National Academy of Sciences of the United States of America; (USA) Vol. 87:1; ISSN 0027-8424; ISSN PNASA
• Country of Publication: United States
• Language: English

Related Subjects

550201* -- Biochemistry-- Tracer Techniques
59 BASIC BIOLOGICAL SCIENCES
ANIMAL CELLS
ANIMALS
BETA DECAY RADIOISOTOPES
BETA-MINUS DECAY RADIOISOTOPES
BONE MARROW CELLS
CONNECTIVE TISSUE CELLS
DAYS LIVING RADIOISOTOPES
DNA HYBRIDIZATION
ELECTROPHORESIS
ENZYMES
GENE RECOMBINATION
GENE REGULATION
HYBRIDIZATION
HYDROLASES
ISOTOPES
LIGHT NUCLEI
MAMMALS
MICE
MICROORGANISMS
NON-PEPTIDE C-N HYDROLASES
NUCLEI
ODD-ODD NUCLEI
ONCOGENIC VIRUSES
PARASITES
PHOSPHORUS 32
PHOSPHORUS ISOTOPES
RADIOISOTOPES
RODENTS
SOMATIC CELLS
STEM CELLS
VERTEBRATES
VIRUSES