Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements

β-globin lentiviral vectors (β-LV) have faced challenges in clinical translation for gene therapy of sickle cell disease (SCD) due to low titer and sub-optimal gene transfer to hematopoietic stem and progenitor cells (HSPCs). To overcome the challenge of preserving efficacious expression while incre...

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Bibliographic Details
Published in:Mol Ther
Main Authors: Morgan, Richard A., Unti, Mildred J., Aleshe, Bamidele, Brown, Devin, Osborne, Kyle S., Koziol, Colin, Ayoub, Paul G., Smith, Oliver B., O’Brien, Rachel, Tam, Curtis, Miyahira, Eric, Ruiz, Marlene, Quintos, Jason P., Senadheera, Shantha, Hollis, Roger P., Kohn, Donald B.
Format: Artigo
Language:Inglês
Published: American Society of Gene & Cell Therapy 2020
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6953778/
https://ncbi.nlm.nih.gov/pubmed/31628051
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.09.020
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