The New Self-Inactivating Lentiviral Vector for Thalassemia Gene Therapy Combining Two HPFH Activating Elements Corrects Human Thalassemic Hematopoietic Stem Cells

To address how low titer, variable expression, and gene silencing affect gene therapy vectors for hemoglobinopathies, in a previous study we successfully used the HPFH (hereditary persistence of fetal hemoglobin)-2 enhancer in a series of oncoretroviral vectors. On the basis of these data, we genera...

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Détails bibliographiques
Auteurs principaux: Papanikolaou, Eleni, Georgomanoli, Maria, Stamateris, Evangelos, Panetsos, Fottes, Karagiorga, Markisia, Tsaftaridis, Panagiotis, Graphakos, Stelios, Anagnou, Nicholas P.
Format: Artigo
Langue:Inglês
Publié: Mary Ann Liebert, Inc. 2012
Sujets:
Research Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3260446/
https://ncbi.nlm.nih.gov/pubmed/21875313
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.048
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