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.
作者信息
Patsali Petros, Turchiano Giandomenico, Papasavva Panayiota, Romito Marianna, Loucari Constantinos C, Stephanou Coralea, Christou Soteroulla, Sitarou Maria, Mussolino Claudio, Cornu Tatjana I, Antoniou Michael N, Lederer Carsten W, Cathomen Toni, Kleanthous Marina
机构信息
Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
Department of Medical and Molecular Genetics, King's College London, London, UK.
出版信息
Haematologica. 2019 Nov;104(11):e497-e501. doi: 10.3324/haematol.2018.215178. Epub 2019 Apr 19.
Abstract
摘要
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