Carsten W. Lederer

Associate Professor

Current Position

Associate Scientist, Molecular Genetics Thalassaemia Department, The Cyprus Institute of Neurology & Genetics

Associate Professor, Cyprus School of Molecular Medicine

CSMM Activities

Course Coordinator of the Preparatory Course: Introduction to Molecular Biomedical Sciences

Faculty of the main course: MM101: Molecular Basis of Monogenic Diseases

Coordinator of the Transferrable Skills courses and the CING Lectures site seminar series

Research Interests

Current research focus is the gene therapy of β-haemoglobinopathies and particularly of β-thalassaemia by three different approaches.

  • Mutation-specific RNAi-supplementation of gene addition.

This strategy employs lentiviral expression vector for permanent gene addition in cell lines and in primary haematopoietic stem and progenitor cells. See e.g. Patsali et al. 2018.

  • Genome editing of disease modifiers.

This strategy is based on the removal or modification of γ-globin repressors to activate the β-globin-like γ-globin gene and employs designer nucleases and base editors, delivered by lentiviral expression vectors or by electroporation of mRNAs (for TALEN) or ribonucleoprotein complexes (for CRISPR/Cas). See e.g. Loucari et al. 2018.

  • Homology-independent gene repair.

This strategy is based on the removal of aberrant regulatory elements and employs designer nucleases and base editors, delivered by plasmid transfection or lentiviral transduction for proof of principle and by electroporation of mRNAs (for TALEN) or of ribonucleoprotein complexes (for CRISPR/Cas) towards clinical translation. See e.g. Patsali et al. 2019, Patsali et al. 2019, Patsali et al. 2019, Papasavva et al. 2019.


Dr Lederer received his PhD from the University of East Anglia for work in plant virology at the John Innes Centre, Norwich, UK. He is now a group leader at the Molecular Genetics Thalassaemia Department (MGTD; head: Marina Kleanthous) of the Cyprus Institute of Neurology and Genetics (CING), where he heads the MGTD Gene Therapy and Editing unit. Dr Lederer is Associate Professor and course coordinator at the Cyprus School for Molecular Medicine, associate editor of Frontiers in Genome Editing, executive board member of the Global Globin 2020 Challenge, curator of the ITHANET Portal, member of the ClinGen-recognised draft Expert Panel for Haemoglobinopathies, vice president of the Cyprus Society of Human Genetics and a member of the European Society of Gene and Cell Therapy and the Hellenic Society of Gene Therapy and Regenerative Medicine


Dr Lederer is author of 39 peer-reviewed articles with over 2100 literature citations and an h index of 17 (i10 index 23). For an updated list of publications, see here.

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