CRISPR/Cas9-modified Hematopoietic Stem Cells-present and Future Perspectives for Stem Cell Transplantation

Overview

Journal Bone Marrow Transplant

Specialty General Surgery

Date 2019 Mar 24

PMID 30903024

Citations 12

Authors

Alberto Daniel-Moreno

Andres Lamsfus-Calle

Janani Raju

Justin S Antony

Rupert Handgretinger

Markus Mezger

Affiliations

Soon will be listed here.

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) is a standard therapeutic intervention for hematological malignancies and several monogenic diseases. However, this approach has limitations related to lack of a suitable donor, graft-versus-host disease and infectious complications due to immune suppression. On the contrary, autologous HSCT diminishes the negative effects of allogeneic HSCT. Despite the good efficacy, earlier gene therapy trials with autologous HSCs and viral vectors have raised serious safety concerns. However, the CRISPR/Cas9-edited autologous HSCs have been proposed to be an alternative option with a high safety profile. In this review, we summarized the possibility of CRISPR/Cas9-mediated autologous HSCT as a potential treatment option for various diseases supported by preclinical gene-editing studies. Furthermore, we discussed future clinical perspectives and possible clinical grade improvements of CRISPR/cas9-mediated autologous HSCT.

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