Disruptive Technology: CRISPR/Cas-Based Tools and Approaches

por Patsali, Petros, Kleanthous, Marina, Lederer, Carsten W.
Publicado 2019

Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

por Papasavva, Panayiota, Kleanthous, Marina, Lederer, Carsten W.
Publicado 2019

Therapy Development by Genome Editing of Hematopoietic Stem Cells

por Koniali, Lola, Lederer, Carsten W., Kleanthous, Marina
Publicado 2021

Pathways and genes differentially expressed in the motor cortex of patients with sporadic amyotrophic lateral sclerosis

por Lederer, Carsten W, Torrisi, Antonietta, Pantelidou, Maria, Santama, Niovi, Cavallaro, Sebastiano
Publicado 2007

IthaGenes: An Interactive Database for Haemoglobin Variations and Epidemiology

por Kountouris, Petros, Lederer, Carsten W., Fanis, Pavlos, Feleki, Xenia, Old, John, Kleanthous, Marina
Publicado 2014

Selection and Identification of Skeletal-Muscle-Targeted RNA Aptamers

por Philippou, Styliana, Mastroyiannopoulos, Nikolaos P., Makrides, Neoklis, Lederer, Carsten W., Kleanthous, Marina, Phylactou, Leonidas A.
Publicado 2018

Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBB(IVSI−110 (G > A)) β-Thalassemia

por Patsali, Petros, Papasavva, Panayiota, Christou, Soteroulla, Sitarou, Maria, Antoniou, Michael N., Lederer, Carsten W., Kleanthous, Marina
Publicado 2020

Recent trends in the gene therapy of β-thalassemia

por Finotti, Alessia, Breda, Laura, Lederer, Carsten W, Bianchi, Nicoletta, Zuccato, Cristina, Kleanthous, Marina, Rivella, Stefano, Gambari, Roberto
Publicado 2015

CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation

por Papasavva, Panayiota L., Patsali, Petros, Loucari, Constantinos C., Kurita, Ryo, Nakamura, Yukio, Kleanthous, Marina, Lederer, Carsten W.
Publicado 2022

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

por Drakopoulou, Ekati, Georgomanoli, Maria, Lederer, Carsten W., Panetsos, Fottes, Kleanthous, Marina, Voskaridou, Ersi, Valakos, Dimitrios, Papanikolaou, Eleni, Anagnou, Nicholas P.
Publicado 2022

Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells

por Papasavva, Panayiota L., Papaioannou, Nikoletta Y., Patsali, Petros, Kurita, Ryo, Nakamura, Yukio, Sitarou, Maria, Christou, Soteroulla, Kleanthous, Marina, Lederer, Carsten W.
Publicado 2021

Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies

por Lederer, Carsten W., Koniali, Lola, Buerki-Thurnherr, Tina, Papasavva, Panayiota L., La Grutta, Stefania, Licari, Amelia, Staud, Frantisek, Bonifazi, Donato, Kleanthous, Marina
Publicado 2022

High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis

por Papaioannou, Nikoletta Y., Patsali, Petros, Naiisseh, Basma, Papasavva, Panayiota L., Koniali, Lola, Kurita, Ryo, Nakamura, Yukio, Christou, Soteroula, Sitarou, Maria, Mussolino, Claudio, Cathomen, Toni, Kleanthous, Marina, Lederer, Carsten W.
Publicado 2023

Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies

por Loucari, Constantinos C., Patsali, Petros, van Dijk, Thamar B., Stephanou, Coralea, Papasavva, Panayiota, Zanti, Maria, Kurita, Ryo, Nakamura, Yukio, Christou, Soteroulla, Sitarou, Maria, Philipsen, Sjaak, Lederer, Carsten W., Kleanthous, Marina
Publicado 2018

Adapting the ACMG/AMP variant classification framework: A perspective from the ClinGen Hemoglobinopathy Variant Curation Expert Panel

por Kountouris, Petros, Stephanou, Coralea, Lederer, Carsten W., Traeger‐Synodinos, Joanne, Bento, Celeste, Harteveld, Cornelis L., Fylaktou, Eirini, Koopmann, Tamara T., Halim‐Fikri, Hashim, Michailidou, Kyriaki, Nfonsam, Landry E., Waye, John S., Zilfalil, Bin A., Kleanthous, Marina
Publicado 2021

Sex-specific transcriptional profiles identified in β-thalassemia patients

por Nanou, Aikaterini, Toumpeki, Chrisavgi, Fanis, Pavlos, Bianchi, Nicoletta, Cosenza, Lucia Carmela, Zuccato, Cristina, Sentis, George, Giagkas, Giorgos, Stephanou, Coralea, Phylactides, Marios, Christou, Soteroula, Hadjigavriel, Michalis, Sitarou, Maria, Lederer, Carsten W., Gambari, Roberto, Kleanthous, Marina, Katsantoni, Eleni
Publicado 2021

The molecular spectrum and distribution of haemoglobinopathies in Cyprus: a 20-year retrospective study

por Kountouris, Petros, Kousiappa, Ioanna, Papasavva, Thessalia, Christopoulos, George, Pavlou, Eleni, Petrou, Miranda, Feleki, Xenia, Karitzie, Eleni, Phylactides, Marios, Fanis, Pavlos, Lederer, Carsten W., Kyrri, Andreani R., Kalogerou, Eleni, Makariou, Christiana, Ioannou, Christiana, Kythreotis, Loukas, Hadjilambi, Georgia, Andreou, Nicoletta, Pangalou, Evangelia, Savvidou, Irene, Angastiniotis, Michael, Hadjigavriel, Michael, Sitarou, Maria, Kolnagou, Annita, Kleanthous, Marina, Christou, Soteroula
Publicado 2016

Global Globin Network Consensus Paper: Classification and Stratified Roadmaps for Improved Thalassaemia Care and Prevention in 32 Countries

por Halim-Fikri, Bin Hashim, Lederer, Carsten W., Baig, Atif Amin, Mat-Ghani, Siti Nor Assyuhada, Syed-Hassan, Sharifah-Nany Rahayu-Karmilla, Yusof, Wardah, Abdul Rashid, Diana, Azman, Nurul Fatihah, Fucharoen, Suthat, Panigoro, Ramdan, Silao, Catherine Lynn T., Viprakasit, Vip, Jalil, Norunaluwar, Mohd Yasin, Norafiza, Bahar, Rosnah, Selvaratnam, Veena, Mohamad, Norsarwany, Nik Hassan, Nik Norliza, Esa, Ezalia, Krause, Amanda, Robinson, Helen, Hasler, Julia, Stephanou, Coralea, Raja-Sabudin, Raja-Zahratul-Azma, Elion, Jacques, El-Kamah, Ghada, Coviello, Domenico, Yusoff, Narazah, Abdul Latiff, Zarina, Arnold, Chris, Burn, John, Kountouris, Petros, Kleanthous, Marina, Ramesar, Raj, Zilfalil, Bin Alwi
Publicado 2022

Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi

por Cavazza, Alessia, Hendel, Ayal, Bak, Rasmus O., Rio, Paula, Güell, Marc, Lainšček, Duško, Arechavala-Gomeza, Virginia, Peng, Ling, Hapil, Fatma Zehra, Harvey, Joshua, Ortega, Francisco G., Gonzalez-Martinez, Coral, Lederer, Carsten W., Mikkelsen, Kasper, Gasiunas, Giedrius, Kalter, Nechama, Gonçalves, Manuel A.F.V., Petersen, Julie, Garanto, Alejandro, Montoliu, Lluis, Maresca, Marcello, Seemann, Stefan E., Gorodkin, Jan, Mazini, Loubna, Sanchez, Rosario, Rodriguez-Madoz, Juan R., Maldonado-Pérez, Noelia, Laura, Torella, Schmueck-Henneresse, Michael, Maccalli, Cristina, Grünewald, Julian, Carmona, Gloria, Kachamakova-Trojanowska, Neli, Miccio, Annarita, Martin, Francisco, Turchiano, Giandomenico, Cathomen, Toni, Luo, Yonglun, Tsai, Shengdar Q., Benabdellah, Karim
Publicado 2023