Paper of the field n°8
Click here to read more about the next-generation in vivo gene editing of human hematopoietic stem and progenitor cells! Abstract for [...]
Publications
EDITSCD results will be published and made freely available to the entire scientific and public communities during the course of the project and further. In addition, topic-related publications and news will be published regularly int EDITSCD website.
Kalter, Nechama, et al. "Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges". https://doi.org/10.1016/j.omtn.2025.102636
EDITSCD
Cullot, Grégoire, et al. « Genome Editing with the HDR-Enhancing DNA-PKcs Inhibitor AZD7648 Causes Large-Scale Genomic Alterations ». Nature Biotechnology, vol. 43, [...]
EDITSCD
Venkatesan, Vigneshwaran, et al. « Editing the Core Region in HPFH Deletions Alters Fetal and Adult Globin Expression for Treatment of [...]
EDITSCD
Dacquay, Louis C., et al. « Dual Inhibition of DNA-PK and Polϴ Boosts Precision of Diverse Prime Editing Systems ». Nature Communications, [...]
EDITSCD
EDITSCD & ERN-EuroBloodNet: Focus on Genetic Therapy for People Living with SCD
ERN-EuroBloodNet and the EDITSCD Consortium are launching a series of webinars aimed at providing an overview of gene therapy and [...]
EDITSCD
Sobrino, Steicy, et al. « Severe Inflammation and Lineage Skewing Are Associated with Poor Engraftment of Engineered Hematopoietic Stem Cells in [...]
EDITSCD
Paper of the field n°7
Click here to read more about the new method that speeds up gene editing by using packaged CRISPR–Cas9 tools.! Abstract for [...]
Brusson, Megane, et Annarita Miccio. « Une approche CRISPR/Cas pour traiter les β-hémoglobinopathies ». médecine/sciences, vol. 41, no 1, janvier 2025, p. [...]
EDITSCD
Frati, Giacomo, et al. « Safety and Efficacy Studies of CRISPR-Cas9 Treatment of Sickle Cell Disease Highlights Disease-Specific Responses ». Molecular Therapy: [...]
EDITSCD
Paper of the field n°6
Click here to read more about the genome engineering with Cas9 and adeno-associated virus strategies! Abstract for General public Genome editing using [...]
An anonymous survey for Sickle Cell Disease community
For World Sickle Cell Day 2024, ERN-EuroBloodNet is gathering the voices of people living with Sickle Cell Disease globally via [...]
Paper of the field n°5
Click here to read more about the need to personalize genome-editing experiments! Abstract for General public Variations within the human genome affect [...]
EDITSCD consortium grows!
The Institute of Bioorganic Chemistry of the Polish Academy of Sciences has joined the project! As part of the "Hop-on facility" [...]
Wang, Jinglong, et al. « DNA-PKcs Suppresses Illegitimate Chromosome Rearrangements ». Nucleic Acids Research, février 2024, p. gkae140. https://doi.org/10.1093/nar/gkae140
Paper of the field n°4
Click here to read more about the latest groundbreaking ex vivo prime editing results! [...]
Wimberger, Sandra, et al. « Simultaneous Inhibition of DNA-PK and Polϴ Improves Integration Efficiency and Precision of Genome Editing ». Nature Communications, [...]
EDITSCD
Paper of the field n°3
Click to find how targeting the bone marrow niche could improve gene therapy outcome! [...]
First CRISPR Gene Editing Treatment for Sickle Cell Disease
In the history of sickle-cell anaemia, UK and FDA approve gene therapy for the first time! It's a real game [...]
Sighinolfi, Silvia, et al. « Intracellular Iron Controls HSC Metabolism By Affecting Mitochondrial Fitness ». Blood, vol. 142, no Supplement 1, novembre [...]
EDITSCD
Paper of the field n°2
Click to find out how the regulation of p53 and cell proliferation during editing can contribute to the development of [...]
P, Zarghamian, et al. « Clinical Genome Editing to Treat Sickle Cell Disease-A Brief Update ». Frontiers in Medicine, vol. 9, janvier [...]
EDITSCD
Antoniou, Panagiotis, et al. « Base-Editing-Mediated Dissection of a γ-Globin Cis-Regulatory Element for the Therapeutic Reactivation of Fetal Hemoglobin Expression ». Nature Communications, [...]
