Luigi Naldini

TL;DR: Hematopoietic stem-cell gene therapy has proven to be an effective treatment for several primary immunodeficiencies, and yet companies in this space are withdrawing from the EU market.

TL;DR: A gene vector adapted for transient expression of a transgene in a peripheral organ cell comprising a regulatory sequence operably linked to a trans-gene wherein the regulatory sequence prevents or reduces expression of said transgenes in hematopoietic lineage cells as discussed by the authors.

TL;DR: Investigation of factors influencing the extent of lentiviral vector (LV) shedding upon ex vivo transduction of human hematopoietic stem and progenitor cells indicates that, although vector carry-over is detectable when using laboratory-grade vector stocks, the use of clinical- grade vector stocks strongly decreases the amount of inadvertentTransduction of secondary targets, likely because of the higher degree of purification.

TL;DR: In this paper , the authors show that mobilizers create an opportunity for seamless engraftment of exogenous cells, which effectively outcompete those mobilized, to repopulate the depleted bone marrow.

TL;DR: An optimized laboratory-scale workflow whereby an LV-containing supernatant is purified and concentrated by sequential chromatographic steps, resulting in higher gene transfer and editing efficiency in severe combined immunodeficiency-repopulating hematopoietic stem and progenitor cells (HSPCs) ex vivo, as well as reduced activation of inflammatory responses ex vivo and in vivo as compared to TU-matched, laboratory-grade vectors.