Ospedale San Raffaele S.r.l.

Fondazione Telethon

Fondazione Telethon Ets

Bernhard Rudolf Gentner

Luigi Naldini

Alessandra Biffi

Pietro Genovese

Anna Christina Kajaste-rudnitski

Giulia Schiroli

Carolina Petrillo

Erika Zonari

Francesco Boccalatte

METHOD FOR IMPROVING RETROVIRAL TRANSDUCTION AND GENE EDITING IN HEMATOPOIETIC STEM CELLS USING CLYCLOSPORIN H AND UM171

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.

Method for improving retroviral transduction and gene editing in hematopoietic stem cells using cyclosporine h (CsH)

A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell.

Gene vector

A method of preparing a therapeutic cell population for clinical use from a starting population of cells comprising haematopoietic stem cells, said method comprising separating a population of cells that substantially do not express CD38 but which express CD34 from the starting population of cells, and transducing the separated cell population with a vector to obtain the therapeutic cell population.

Methods for genetic modification of stem cells

Growing community of inventors

Milan, Italy

Bernhard Rudolf Gentner