Expression of the human BETA-globin gene after retroviral transfer into murine erythroleukemia cells and human BFU-E cells

Journal Article · Thu Mar 31 23:00:00 EST 1988 · Mol. Cell. Biol.; (United States)

DOI:https://doi.org/10.1128/MCB.8.4.1725· OSTI ID:6945053

Bender, M A; Miller, A D; Gelinas, R E

Replication-defective amphotropic retrovirus vectors containing either the human ..beta..-globin gene with introns or an intronless ..beta..-globin minigene were constructed and used to study ..beta..-globin expression following gene transfer into hematopoietic cells. The ..beta..-globin genes were marked by introducing a 6-base-pair insertion into the region corresponding to the 5' untranslated region of the ..beta..-globin mRNA to allow detection of RNA encoded by the new gene in human cells expressing normal human ..beta..-globin RNA. Introduction of a virus containing the ..beta..-globin gene with introns into murine erythroleukemia cells resulted in inducible expression of human ..beta..-globin RNA and protein, while the viruses containing the minigene were inactive. The introduced human ..beta..-globin gene was 6 to 110% as active as the endogenous mouse ..beta../sup maj/-globin genes in six randomly chosen cell clones. Introduction of the viruses into human BFU-E cells, followed by analysis of marked and unmarked globin RNAs in differentiated erythroid colonies, revealed that the introduced ..beta..-globin gene was about 5% as active as the endogenous genes in these normal human erythroid cells and that again the minigene was inactive. These data are discussed in terms of the potential treatment of genetic disease by gene therapy.

• Research Organization: Dept. of Molecular Medicine, Fred Hutchinson Cancer Research Center, 1124 Columbia Street, Seattle, WA (US)
• OSTI ID: 6945053
• Journal Information: Mol. Cell. Biol.; (United States), Journal Name: Mol. Cell. Biol.; (United States) Vol. 8:4; ISSN MCEBD
• Country of Publication: United States
• Language: English

Related Subjects

550400* -- Genetics
59 BASIC BIOLOGICAL SCIENCES
ANIMALS
BIOLOGY
DEFECTS
DISEASES
DNA REPLICATION
GENES
GENETIC ENGINEERING
GENETICS
GLOBIN
HEMIC DISEASES
HEREDITARY DISEASES
LEUKEMIA
MALES
MAMMALS
MAN
MEN
MESSENGER-RNA
MICE
MICROORGANISMS
NEOPLASMS
NUCLEIC ACID REPLICATION
NUCLEIC ACIDS
ONCOGENIC VIRUSES
ORGANIC COMPOUNDS
PARASITES
PHENOTYPE
PRIMATES
PROTEINS
RNA
RODENTS
THERAPY
VERTEBRATES
VIRUSES