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US 12,485,187 B2Granted
Method for improving retroviral transduction and gene editing in hematopoietic stem cells using clyclosporin H and UM171
AssigneesOspedale San Raffaele S.R.L., Fondazione Telethon
InventorsAnna Christina Kajaste-Rudnitski, Carolina Petrillo, Bernhard Rudolf Gentner, Luigi Naldini, Pietro Genovese, Giulia Schiroli
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Abstract
Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.
Claims22
A method of transducing a population of CD34+ HSPCs comprising the steps of:
- (a) culturing the population of HSPCs in the presence of UM171;
- (b) contacting the population of HSPCs with cyclosporin H (CsH);
- and
- (c) transducing the population of HSPCs with a viral vector, wherein the viral vector is a lentiviral or gammaretroviral vector, and wherein the viral vector is pseudotyped to enter cells via an endocytosis-dependent mechanism.
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