Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

Rajeev Rai; Marianna Romito; Elizabeth Rivers; Giandomenico Turchiano; Georges Blattner; Winston Vetharoy; Dariusz Ladon; Geoffroy Andrieux; Fang Zhang; Marta Zinicola; Diego Leon-Rico; Giorgia Santilli; Adrian J. Thrasher; Alessia Cavazza

doi:10.1038/s41467-020-17626-2

Nature Communications (Aug 2020)

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

Rajeev Rai,
Marianna Romito,
Elizabeth Rivers,
Giandomenico Turchiano,
Georges Blattner,
Winston Vetharoy,
Dariusz Ladon,
Geoffroy Andrieux,
Fang Zhang,
Marta Zinicola,
Diego Leon-Rico,
Giorgia Santilli,
Adrian J. Thrasher,
Alessia Cavazza

Affiliations

Rajeev Rai: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Marianna Romito: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Elizabeth Rivers: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Giandomenico Turchiano: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Georges Blattner: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Winston Vetharoy: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Dariusz Ladon: SIHMDS-Acquired Genomics, Great Ormond Street Hospital for Children NHS Foundation Trust
Geoffroy Andrieux: Institute of Medical Bioinformatics and System Medicine, University of Freiburg
Fang Zhang: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Marta Zinicola: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Diego Leon-Rico: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Giorgia Santilli: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Adrian J. Thrasher: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London
Alessia Cavazza: Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London

DOI: https://doi.org/10.1038/s41467-020-17626-2
Journal volume & issue: Vol. 11, no. 1
pp. 1 – 15

Abstract

Read online

In recent years, hematopoietic stem cells gene editing has emerged as a promising tool to treat blood disorders. Here the authors develop a CRISPR/Cas9-based genome editing strategy that allows the precise correction of Wiskott-Aldrich Syndrome in vitro and in vivo with high efficiency.

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/

About the journal