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Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas- and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells

Patsali, P; Turchiano, G; Papasavva, P; Romito, M; Loucari, CC; Stephanou, C; Christou, S; ... Kleanthous, M; + view all (2019) Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas- and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells. Haematologica 10.3324/haematol.2018.215178. (In press). Green open access

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Type: Article
Title: Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas- and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells
Location: Italy
Open access status: An open access version is available from UCL Discovery
DOI: 10.3324/haematol.2018.215178
Publisher version: https://doi.org/10.3324/haematol.2018.215178
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Gene Therapy and Transfer, Gene editing, Hematopoietic Stem Cell, Splicing, Thalassemia
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10074222
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