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

Francesca Tucci; Stefania Galimberti; Luigi Naldini; Maria Grazia Valsecchi; Alessandro Aiuti

doi:10.1038/s41467-022-28762-2

Nature Communications (Mar 2022)

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

Francesca Tucci,
Stefania Galimberti,
Luigi Naldini,
Maria Grazia Valsecchi,
Alessandro Aiuti

Affiliations

Francesca Tucci: Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute
Stefania Galimberti: Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano — Bicocca
Luigi Naldini: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute
Maria Grazia Valsecchi: Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano — Bicocca
Alessandro Aiuti: Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute

DOI: https://doi.org/10.1038/s41467-022-28762-2
Journal volume & issue: Vol. 13, no. 1
pp. 1 – 13

Abstract

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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.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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