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. 2019 Nov;104(11):e497-e501. doi: 10.3324/haematol.2018.215178. Epub 2019 Apr 19.

Petros Patsali^{1

2}, Giandomenico Turchiano^{3

4}, Panayiota Papasavva^{1

5}, Marianna Romito^{3

4}, Constantinos C Loucari^{1

5}, Coralea Stephanou^{1

2}, Soteroulla Christou⁶, Maria Sitarou⁶, Claudio Mussolino^{3

4}, Tatjana I Cornu^{3

4}, Michael N Antoniou², Carsten W Lederer^{7

5}, Toni Cathomen^{8

4

9}, Marina Kleanthous^{10

5}

¹ Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
² Department of Medical and Molecular Genetics, King's College London, London, UK.
³ Institute for Transfusion Medicine and Gene Therapy, Medical Center, University of Freiburg, Freiburg, Germany.
⁴ Center for Chronic Immunodeficiency, Medical Center, University of Freiburg, Freiburg, Germany.
⁵ Cyprus School of Molecular Medicine, Nicosia, Cyprus.
⁶ Thalassemia Center, Cyprus Ministry of Health, Cyprus.
⁷ Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus lederer@cing.ac.cy.
⁸ Institute for Transfusion Medicine and Gene Therapy, Medical Center, University of Freiburg, Freiburg, Germany toni.cathomen@uniklinik-freiburg.de.
⁹ Faculty of Medicine, University of Freiburg, Freiburg, Germany.
¹⁰ Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus marinakl@cing.ac.cy.

No abstract available

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