Genetic correction of sickle cell disease: Insights using transgenic mouse models
Autor: | A Wright, Marie-José Blouin, C N Ou, M Sorette, Hugues Beauchemin, M.E. De Paepe, B Nakamoto, A M Bleau, George Stamatoyannopoulos, Marie Trudel |
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Rok vydání: | 2000 |
Předmět: |
Genetically modified mouse
Anemia Transgene Genetic enhancement Longevity Cell Mice Transgenic Anemia Sickle Cell Genetic Therapy General Medicine Biology medicine.disease General Biochemistry Genetics and Molecular Biology Disease Models Animal Mice medicine.anatomical_structure In vivo Fetal hemoglobin Immunology medicine Animals Erythropoiesis RNA Messenger Hemoglobin Fetal Hemoglobin |
Zdroj: | Nature Medicine. 6:177-182 |
ISSN: | 1546-170X 1078-8956 |
Popis: | Sickle cell disease is a hereditary disorder characterized by erythrocyte deformity due to hemoglobin polymerization. We assessed in vivo the potential curative threshold of fetal hemoglobin in the SAD transgenic mouse model of sickle cell disease using mating with mice expressing the human fetal Agamma-globin gene. With increasing levels of HbF, AgammaSAD mice showed considerable improvement in all hematologic parameters, morphopathologic features and life span/survival. We established the direct therapeutic effect of fetal hemoglobin on sickle cell disease and demonstrated correction by increasing fetal hemoglobin to about 9-16% in this mouse model. This in vivo study emphasizes the potential of the SAD mouse models for quantitative analysis of gene therapy approaches. |
Databáze: | OpenAIRE |
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