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Review
. 2023 May 4;30(5):549-570.
doi: 10.1016/j.stem.2023.04.014.

Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

Samuele Ferrari  1 , Erika Valeri  1 , Anastasia Conti  1 , Serena Scala  1 , Annamaria Aprile  1 , Raffaella Di Micco  1 , Anna Kajaste-Rudnitski  1 , Eugenio Montini  1 , Giuliana Ferrari  2 , Alessandro Aiuti  2 , Luigi Naldini  3
Affiliations
Free article
Review

Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

Samuele Ferrari et al. Cell Stem Cell. .
Free article

Abstract

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy (GT) relies on the development of viral vectors as portable "Trojan horses" for safe and efficient gene transfer. The recent advent of novel technologies enabling site-specific gene editing is broadening the scope and means of GT, paving the way to more precise genetic engineering and expanding the spectrum of diseases amenable to HSPC-GT. Here, we provide an overview of state-of-the-art and prospective developments of the HSPC-GT field, highlighting how advances in biological characterization and manipulation of HSPCs will enable the design of the next generation of these transforming therapeutics.

Keywords: CRISPR-Cas; Gene editing; Gene therapy; Hematopoietic stem cells; Primary immunodeficiencies.

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Conflict of interest statement

Declaration of interests L.N. is an inventor on patents on LV technology and gene editing filed by the Salk Institute, Cell Genesys, Telethon Foundation, and/or San Raffaele Scientific Institute. S.F., A.C., R.D.M., and A.K.R. are inventors of patents on gene editing, and E.M. is an inventor of patent on clonal tracking, owned and managed by the San Raffaele Scientific Institute and Telethon Foundation. L.N. is a founder, equity owner, consultant, and member of the scientific advisory board of Genenta Science, a biotechnology company aiming at developing cancer gene therapy by tumor-infiltrating monocytes; GeneSpire, a biotechnology startup developing LV-based liver gene transfer and hematopoietic cell gene editing; and Epsilen Bio/Chroma Medicine, a company developing epigenetic editing. A. Aiuti is a PI of clinical trials sponsored by Orchard Therapeutics, which licensed several gene therapy products originally developed at SR-Tiget. A. Aiuti is a member of the EU Committee for Advanced Therapies (CAT), and his views are personal and may not be understood or quoted as being made on behalf of the European Medicines Agency (EMA). All other authors declare no competing interests.

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