Publications by authors named "Carsten W Lederer"

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HELIOS Action: Advancing research, education, and equity in hemoglobinopathies across Europe and beyond.
Sotiroula Chatzimatthaiou, Fedele Bonifazi, Anna C Gimbert, Raffaella Colombatti, Francesco Cremonesi, Carsten Werner Lederer

Hemasphere· December 2025

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Proceedings of the second annual meeting of GenE-HumDi (COST Action 21113).
María Ortiz-Bueno, Iris Ramos-Hernández, Luis Algeciras-Jiménez, Nechama Kalter, Juan Roberto Rodríguez-Madoz, Carsten Werner Lederer

Front Genome Ed· November 2025

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Above the cut: HbF induction by multiplex prime editing.
Carsten W Lederer

Blood· November 2025

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Evaluation of Five Plasma miRNAs as Biomarkers for Minimally Invasive Staging of Liver Fibrosis in β-Thalassaemia Patients.
Sevgi Özkaramehmet, Savanna Andreou, Kristia Yiangou, Soteroula Christou, Michalis Hadjigavriel, Carsten W Lederer

Int J Mol Sci· September 2025

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Genetic Modifiers Associated with Vaso-Occlusive Crises and Acute Pain Phenomena in Sickle Cell Disease: A Scoping Review.
Froso Sophocleous, Natasha M Archer, Carsten W Lederer,

Int J Mol Sci· May 2025

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Non-Invasive Determination of the Paternal Inheritance in Pregnancies at Risk for β-Thalassaemia by Analyzing Cell-Free Fetal DNA Using Targeted Next-Generation Sequencing.
Stefania Byrou, Rutger W W Brouwer, Marios Tomazou, Stella Tamana, Petros Kountouris, Carsten W Lederer

Int J Mol Sci· January 2025

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

Front Genet· November 2024

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Global Globin Network and adopting genomic variant database requirements for thalassemia.
Hashim Halim-Fikri, Ninie Nadia Zulkipli, Hafiza Alauddin, Celeste Bento, Carsten W Lederer

Database (Oxford)· September 2024

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Context base editing for splice correction of IVSI-110 β-thalassemia.
Basma Naiisseh, Panayiota L Papasavva, Nikoletta Y Papaioannou, Marios Tomazou, Lola Koniali, Carsten W Lederer

Mol Ther Nucleic Acids· June 2024

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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Alessia Cavazza, Ayal Hendel, Rasmus O Bak, Paula Rio, Marc Güell, Carsten W Lederer

Mol Ther Nucleic Acids· December 2023

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Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by Gene Addition and Mutation-Specific RNA Interference.
Lola Koniali, Christina Flouri, Markela I Kostopoulou, Nikoletta Y Papaioannou, Panayiota L Papasavva, Carsten W Lederer

Cells· December 2023

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High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on mRNA synthesis.
Nikoletta Y Papaioannou, Petros Patsali, Basma Naiisseh, Panayiota L Papasavva, Lola Koniali, Carsten W Lederer

Front Genome Ed· March 2023

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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.
Ekati Drakopoulou, Maria Georgomanoli, Carsten W Lederer, Fottes Panetsos, Marina Kleanthous

Viruses· December 2022

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Adapting the ACMG/AMP variant classification framework: A perspective from the ClinGen Hemoglobinopathy Variant Curation Expert Panel.
Petros Kountouris, Coralea Stephanou, Carsten W Lederer, Joanne Traeger-Synodinos, Celeste Bento

Hum Mutat· August 2022

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Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.
Carsten W Lederer, Lola Koniali, Tina Buerki-Thurnherr, Panayiota L Papasavva, Stefania La Grutta

Pharmaceutics· April 2022

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Global Globin Network Consensus Paper: Classification and Stratified Roadmaps for Improved Thalassaemia Care and Prevention in 32 Countries.
Bin Hashim Halim-Fikri, Carsten W Lederer, Atif Amin Baig, Siti Nor Assyuhada Mat-Ghani, Sharifah-Nany Rahayu-Karmilla Syed-Hassan

J Pers Med· March 2022

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

Int J Mol Sci· January 2022

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Regulation of ER Composition and Extent, and Putative Action in Protein Networks by ER/NE Protein TMEM147.
Giannis Maimaris, Andri Christodoulou, Niovi Santama, Carsten Werner Lederer

Int J Mol Sci· September 2021

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Editorial: Mutation-Specific Gene Editing for Blood Disorders.
Carsten Werner Lederer, Pietro Genovese, Annarita Miccio, Sjaak Philipsen

Front Genome Ed· September 2021

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

Cells· June 2021

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Sex-specific transcriptional profiles identified in β-thalassemia patients.
Aikaterini Nanou, Chrisavgi Toumpeki, Pavlos Fanis, Nicoletta Bianchi, Lucia Carmela Cosenza, Carsten W Lederer

Haematologica· April 2021

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

Int J Mol Sci· March 2021

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Relative and Absolute Quantification of Aberrant and Normal Splice Variants in β-Thalassemia.
Petros Patsali, Panayiota Papasavva, Soteroulla Christou, Maria Sitarou, Michael N Antoniou, Carsten W Lederer

Int J Mol Sci· September 2020

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TMEM147 interacts with lamin B receptor, regulates its localization and levels, and affects cholesterol homeostasis.
Andri Christodoulou, Giannis Maimaris, Andri Makrigiorgi, Evelina Charidemou, Christian Lüchtenborg, Carsten W Lederer

J Cell Sci· August 2020

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

Int J Mol Sci· February 2020

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