Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia

A primary challenge in lentiviral gene therapy of β-hemoglobinopathies is to maintain low vector copy numbers to avoid genotoxicity while being reliably therapeutic for all genotypes. We designed a high-titer lentiviral vector, LVβ-shα2, that allows coordinated expression of the therapeutic β(A-T87Q...

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Detalles Bibliográficos
Autores principales: Nualkaew, Tiwaporn, Sii-Felice, Karine, Giorgi, Marie, McColl, Bradley, Gouzil, Julie, Glaser, Astrid, Voon, Hsiao P.J., Tee, Hsin Y., Grigoriadis, George, Svasti, Saovaros, Fucharoen, Suthat, Hongeng, Suradej, Leboulch, Philippe, Payen, Emmanuel, Vadolas, Jim
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8417505/
https://www.ncbi.nlm.nih.gov/pubmed/33940155
http://dx.doi.org/10.1016/j.ymthe.2021.04.037

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8417505/
https://www.ncbi.nlm.nih.gov/pubmed/33940155
http://dx.doi.org/10.1016/j.ymthe.2021.04.037

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