Antiviral Immunity and Nucleic Acid Sensing in Haematopoietic Stem Cell Gene Engineering

Overview

Journal Gene Ther

Date 2020 Jul 15

PMID 32661282

Citations 15

Authors

Francesco Piras

Anna Kajaste-Rudnitski

Affiliations

Soon will be listed here.

Abstract

The low gene manipulation efficiency of human hematopoietic stem and progenitor cells (HSPC) remains a major hurdle for sustainable and broad clinical application of innovative therapies for a wide range of disorders. Given that all current and emerging gene transfer and editing technologies are bound to expose HSPC to exogenous nucleic acids and most often also to viral vectors, we reason that host antiviral factors and nucleic acid sensors play a pivotal role in the efficacy of HSPC genetic manipulation. Here, we review recent progress in our understanding of vector-host interactions and innate immunity in HSPC upon gene engineering and discuss how dissecting this crosstalk can guide the development of more stealth and efficient gene therapy approaches in the future.

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