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

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Acid Ceramidase Depletion Impairs Neuronal Survival and Induces Morphological Defects in Neurites Associated with Altered Gene Transcription and Sphingolipid Content by Kalia Kyriakou, Carsten W. Lederer, Marina Kleanthous, Anthi Drousiotou, Anna Malekkou

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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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Relative and Absolute Quantification of Aberrant and Normal Splice Variants in <i>HBB^{IVSI−110 (G > A)}</i> β-Thalassemia by Petros Patsali, Panayiota Papasavva, Soteroulla Christou, Maria Sitarou, Michael N. Antoniou, Carsten W. Lederer, Marina Kleanthous

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CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation by Panayiota L. Papasavva, Petros Patsali, Constantinos C. Loucari, Ryo Kurita, Yukio Nakamura, Marina Kleanthous, Carsten W. Lederer

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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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Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells by Panayiota L. Papasavva, Nikoletta Y. Papaioannou, Petros Patsali, Ryo Kurita, Yukio Nakamura, Maria Sitarou, Soteroulla Christou, Marina Kleanthous, Carsten W. Lederer

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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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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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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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Global Globin Network Consensus Paper: Classification and Stratified Roadmaps for Improved Thalassaemia Care and Prevention in 32 Countries by Bin Hashim Halim-Fikri, Carsten W. Lederer, Atif Amin Baig, Siti Nor Assyuhada Mat-Ghani, Sharifah-Nany Rahayu-Karmilla Syed-Hassan, Wardah Yusof, Diana Abdul Rashid, Nurul Fatihah Azman, Suthat Fucharoen, Ramdan Panigoro, Catherine Lynn T. Silao, Vip Viprakasit, Norunaluwar Jalil, Norafiza Mohd Yasin, Rosnah Bahar, Veena Selvaratnam, Norsarwany Mohamad, Nik Norliza Nik Hassan, Ezalia Esa, Amanda Krause, Helen Robinson, Julia Hasler, Coralea Stephanou, Raja-Zahratul-Azma Raja-Sabudin, Jacques Elion, Ghada El-Kamah, Domenico Coviello, Narazah Yusoff, Zarina Abdul Latiff, Chris Arnold, John Burn, Petros Kountouris, Marina Kleanthous, Raj Ramesar, Bin Alwi Zilfalil, on behalf of the Global Globin Network (GGN)

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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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