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).
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Type: | Article |
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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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