Therapy Development by Genome Editing of Hematopoietic Stem Cells by Lola Koniali, Carsten W. Lederer, Marina Kleanthous

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IthaGenes: an interactive database for haemoglobin variations and epidemiology. by Petros Kountouris, Carsten W Lederer, Pavlos Fanis, Xenia Feleki, John Old, Marina Kleanthous

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Selection and Identification of Skeletal-Muscle-Targeted RNA Aptamers by Styliana Philippou, Nikolaos P. Mastroyiannopoulos, Neoklis Makrides, Carsten W. Lederer, Marina Kleanthous, Leonidas A. Phylactou

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Differential expression of molecular motors in the motor cortex of sporadic ALS by Maria Pantelidou, Spyros E. Zographos, Carsten W. Lederer, Theodore Kyriakides, Michael W. Pfaffl, Niovi Santama

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Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hemat... by Petros Patsali, Panayiota Papasavva, Coralea Stephanou, Soteroulla Christou, Maria Sitarou, Michael N. Antoniou, Carsten W. Lederer, Marina Kleanthous

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Case report: Rethinking NGS analysis in diagnosing Diamond-Blackfan anemia syndrome by Panayiota L. Papasavva, Konstantinos Kaouranis, Stefania Byrou, Constantina G. Constantinou, Iacovou Efrosini, Marina Kleanthous, Carsten W. Lederer, Thessalia Papasavva

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The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34⁺ Cells from Sickle Cell Disease Patients by Ekati Drakopoulou, Maria Georgomanoli, Carsten W. Lederer, Fottes Panetsos, Marina Kleanthous, Ersi Voskaridou, Dimitrios Valakos, Eleni Papanikolaou, Nicholas P. Anagnou

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Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies by Carsten W. Lederer, Lola Koniali, Tina Buerki-Thurnherr, Panayiota L. Papasavva, Stefania La Grutta, Amelia Licari, Frantisek Staud, Donato Bonifazi, Marina Kleanthous

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Context base editing for splice correction of IVSI-110 β-thalassemia by Basma Naiisseh, Panayiota L. Papasavva, Nikoletta Y. Papaioannou, Marios Tomazou, Lola Koniali, Xenia Felekis, Constantina G. Constantinou, Maria Sitarou, Soteroula Christou, Marina Kleanthous, Carsten W. Lederer, Petros Patsali

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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 by Petros Patsali, Giandomenico Turchiano, Panayiota Papasavva, Marianna Romito, Constantinos C. Loucari, Coralea Stephanou, Soteroulla Christou, Maria Sitarou, Claudio Mussolino, Tatjana I. Cornu, Michael N. Antoniou, Carsten W. Lederer, Toni Cathomen, Marina Kleanthous

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High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis by Nikoletta Y. Papaioannou, Petros Patsali, Basma Naiisseh, Panayiota L. Papasavva, Lola Koniali, Ryo Kurita, Yukio Nakamura, Soteroula Christou, Maria Sitarou, Claudio Mussolino, Claudio Mussolino, Toni Cathomen, Toni Cathomen, Marina Kleanthous, Carsten W. Lederer

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Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by <i>HBB^AS3</i> Gene Addition and Mutation-Specific RNA Interference by Lola Koniali, Christina Flouri, Markela I. Kostopoulou, Nikoletta Y. Papaioannou, Panayiota L. Papasavva, Basma Naiisseh, Coralea Stephanou, Anthi Demetriadou, Maria Sitarou, Soteroula Christou, Michael N. Antoniou, Marina Kleanthous, Petros Patsali, Carsten W. Lederer

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Sex-specific transcriptional profiles identified in β-thalassemia patients by Aikaterini Nanou, Chrisavgi Toumpeki, Pavlos Fanis, Nicoletta Bianchi, Lucia Carmela Cosenza, Cristina Zuccato, George Sentis, Giorgos Giagkas, Coralea Stephanou, Marios Phylactides, Soteroula Christou, Michalis Hadjigavriel, Maria Sitarou, Carsten W. Lederer, Roberto Gambari, Marina Kleanthous, Eleni Katsantoni

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P135: THE CLINGEN HEMOGLOBINOPATHY VARIANT CURATION EXPERT PANEL by C Coralea Stephanou, P Petros Kountouris, C Carsten W Lederer, C Celeste Bento, C Cornelis L Hartveld, J Jan Traeger-Synodinos, J John S Waye, Z Zhiyu Peng, I Irene Fylaktou, H Hashim Halim-Fikri, T Tamara T. Koopmann, L Landry Nfonsam, J Jun Sun, F Franck Nzengu-Lukusa, M Michael Angastiniotis, C Catherine Badens, B Bertha Ibarra Cortes, J Johan T. den Dunnen, J Jacques Elion, S Suthat Fucharoen, K Kyriaki Michailidou, T Thessalia Papasavva, A Antonio Piga, R Raj Ramesar, S Swee Lay Thein, L Léon Tshilolo, Z Zilfalil Bin Alwi, M Marina Kleanthous

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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi by Alessia Cavazza, Ayal Hendel, Rasmus O. Bak, Paula Rio, Marc Güell, Duško Lainšček, Virginia Arechavala-Gomeza, Ling Peng, Fatma Zehra Hapil, Joshua Harvey, Francisco G. Ortega, Coral Gonzalez-Martinez, Carsten W. Lederer, Kasper Mikkelsen, Giedrius Gasiunas, Nechama Kalter, Manuel A.F.V. Gonçalves, Julie Petersen, Alejandro Garanto, Lluis Montoliu, Marcello Maresca, Stefan E. Seemann, Jan Gorodkin, Loubna Mazini, Rosario Sanchez, Juan R. Rodriguez-Madoz, Noelia Maldonado-Pérez, Torella Laura, Michael Schmueck-Henneresse, Cristina Maccalli, Julian Grünewald, Gloria Carmona, Neli Kachamakova-Trojanowska, Annarita Miccio, Francisco Martin, Giandomenico Turchiano, Toni Cathomen, Yonglun Luo, Shengdar Q. Tsai, Karim Benabdellah

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