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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Päätekijät: Papanikolaou, Eleni, Georgomanoli, Maria, Stamateris, Evangelos, Panetsos, Fottes, Karagiorga, Markisia, Tsaftaridis, Panagiotis, Graphakos, Stelios, Anagnou, Nicholas P.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Mary Ann Liebert, Inc. 2012
Aiheet:
Research Articles
Linkit: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
Tagit: Lisää tagi
Ei tageja, Lisää ensimmäinen tagi!

Samankaltaisia teoksia