A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

Ex-vivo gene therapy with hematopoietic stem and progenitor cells (HSPCs) is a promising treatment for monogenic diseases. Here the authors report a systematic review and meta-analysis of available evidence assessing clinical outcomes of HSPC gene therapy from clinical trials.

Bibliographic Details
Main Authors: Francesca Tucci, Stefania Galimberti, Luigi Naldini, Maria Grazia Valsecchi, Alessandro Aiuti
Format: Article
Language:English
Published: Nature Portfolio 2022-03-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-022-28762-2
Description
Summary:Ex-vivo gene therapy with hematopoietic stem and progenitor cells (HSPCs) is a promising treatment for monogenic diseases. Here the authors report a systematic review and meta-analysis of available evidence assessing clinical outcomes of HSPC gene therapy from clinical trials.
ISSN:2041-1723