Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.
Gene therapy of severe hemoglobinopathies will require high-level expression of a transferred globin gene in erythroid cells. Distant regulatory elements flanking the beta-globin gene cluster, the locus control region, are needed for appropriate expression. We have explored the use of a human parvov...
Uloženo v:
| Vydáno v: | Proc Natl Acad Sci U S A |
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| Hlavní autoři: | , , , , , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
National Academy of Sciences
1992
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC49685/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/1323131/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.89.15.7257 |
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