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    Academic Journals

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• Tracking differentiating neural progenitors in pluripotent cultures using microRNA-regulated lentiviral vectors

  Authors:Rohit Sachdeva, Marie E. Jonsson, Jenny Nelander, Agnete Kirkeby, Carolina Guibentif, Bernhard Gentner, et al.From:Proceedings of the National Academy of Sciences of the United States (Vol. 107, Issue 25) Peer-Reviewed

  June 22, 2010136 words Author abstract, Rep...

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• Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

  Authors:Alessandro Fraldi, Marta Serafini, Nicolina Cristina Sorrentino, Bernhard Gentner, Alessandro Aiuti, and Maria Ester BernardoFrom:Italian Journal of Pediatrics (Vol. 44, Issue Suppl 2) Peer-Reviewed

  Nov. 16, 20186,369 words Report 1600LMucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a deficiency in lysosomal enzymes catalyzing the stepwise degradation of glycosaminoglycans (GAGs). The current therapeutic strategies of...

• Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.

  Authors:Bernhard Gentner, Francesca Tucci, Stefania Galimberti, Francesca Fumagalli, Maurizio De Pellegrin, Paolo Silvani, et al.From:The New England Journal of Medicine (Vol. 385, Issue 21) Peer-Reviewed

  Nov. 18, 202134 words Report

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• Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

  Authors:Brian D Brown, Bernhard Gentner, Alessio Cantore, Silvia Colleoni, Mario Amendola, Anna Zingale, et al.From:Nature Biotechnology (Vol. 25, Issue 12) Peer-Reviewed

  Dec. 20079,249 words Article We have shown previously that transgene expression can be suppressed in hematopoietic cells using vectors that are responsive to microRNA (miRNA) regulation. Here we investigate the potential of this approach for more...

• MicroRNA-126--mediated control of cell fate in B-cell myeloid progenitors as a potential alternative to transcriptional factors.

  Authors:Kazuki Okuyama, Tomokatsu Ikawa, Bernhard Gentner, Katsuto Hozumi, Ratanakanit Harnprasopwat, Jun Lu, et al.From:Proceedings of the National Academy of Sciences of the United States (Vol. 110, Issue 33) Peer-Reviewed

  Aug. 13, 2013217 words Report, Author abstr...

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• Targeted genome editing in human repopulating haematopoietic stem cells

  Authors:Pietro Genovese, Giulia Schiroli, Giulia Escobar, Tiziano Di Tomaso, Claudia Firrito, Andrea Calabria, et al.From:Nature (Vol. 510, Issue 7504) Peer-Reviewed

  June 12, 20146,680 words Report 1530LTargeted genome editing by artificial nucleases has brought the goal of site-specific transgene integration and gene correction within the reach of gene therapy. However, its application to long-term repopulating...