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 [...]
In the history of sickle-cell anaemia, UK and FDA approve gene therapy for the first time! It's a real game [...]
Institut Imagine (Paris) will coordinate a Horizon Europe-granted project on genome editing approaches to treat sickle cell disease [...]
Click here to read more about the next-generation in vivo gene editing of human hematopoietic stem and progenitor cells! Abstract for [...]
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
Cullot, Grégoire, et al. « Genome Editing with the HDR-Enhancing DNA-PKcs Inhibitor AZD7648 Causes Large-Scale Genomic Alterations ». Nature Biotechnology, vol. 43, [...]
Venkatesan, Vigneshwaran, et al. « Editing the Core Region in HPFH Deletions Alters Fetal and Adult Globin Expression for Treatment of [...]
ERN-EuroBloodNet and the EDITSCD Consortium is launching the second webinar of the series of webinars regarding gene therapy and genome [...]
Dacquay, Louis C., et al. « Dual Inhibition of DNA-PK and Polϴ Boosts Precision of Diverse Prime Editing Systems ». Nature Communications, [...]
The 3rd edition of the CRISPR MEDiCiNE Conference will take place in Copenhagen, Denmark from April 13th - 16th 2026. Marcello Maresca and Ayal Hendel will give [...]
The 18th annual Sickle Cell in Focus conference will be held in a virtual format and will provide a world-class [...]
Stay tuned! ERN-EuroBloodNet and the EDITSCD Consortium are launching a series of webinars aimed at providing an overview of gene [...]
ERN-EuroBloodNet and the EDITSCD Consortium are launching a series of webinars aimed at providing an overview of gene therapy and [...]
Sobrino, Steicy, et al. « Severe Inflammation and Lineage Skewing Are Associated with Poor Engraftment of Engineered Hematopoietic Stem Cells in [...]
Click here to read more about the new method that speeds up gene editing by using packaged CRISPR–Cas9 tools.! Abstract for [...]
The European Hematology Association (EHA) will host the EHA 2025 Congress in Milan from June 12 to 15, 2025. The scientific [...]
Brusson, Megane, et Annarita Miccio. « Une approche CRISPR/Cas pour traiter les β-hémoglobinopathies ». médecine/sciences, vol. 41, no 1, janvier 2025, p. [...]
Frati, Giacomo, et al. « Safety and Efficacy Studies of CRISPR-Cas9 Treatment of Sickle Cell Disease Highlights Disease-Specific Responses ». Molecular Therapy: [...]
Click here to read more about the genome engineering with Cas9 and adeno-associated virus strategies! Abstract for General public Genome editing using [...]
After three years of project, the Consortium met in person on September 15th and 16th, 2025 to debrief. A summary [...]
A Scientific meeting has been held on November 27th, 2025 on Teams with all Consortium members to present ongoing experiments and updated [...]
A Scientific meeting has been held on May 30th, 2025 on Teams with all Consortium members to present ongoing experiments and updated [...]
A Scientific meeting was held on March 19th, 2025 on Teams with all Consortium members to present ongoing experiments and updated EDITSCD results.
Members of EDITSCD Consortium will join the Synthego Seminar in Paris to discuss sgRNA manufacturing, CRISPR nucleases for therapeutic development [...]
Congress Europe 2024 The World Orphan Drug Congress is the largest and most established orphan drugs & rare diseases meeting of its [...]
The ERN-EuroBloodNet Progress Meetings, are organized every autumn by the ERN-EuroBloodNet Coordination team, to promote networking among the haematologic stakeholders with the aim of identifying needs [...]