![]() Collaborative Prinses Beatrix Spierfonds research project on DMD gene repair –Dr.Gonçalves (LUMC) Financiering voor "Harvey"! - PLN Foundation () Collaborative Dutch Research Council (NWO) - Open Technology Programme research project HARVEY – Prof.Active Horizon Europe Doctoral Network GETRADI - "Gene Therapy of Rare Diseases" Getradi – University of Copenhagen (ku.dk).fellow) winner of the Annual Prize for Neuromuscular Diseases 2022 - Best scientific article Francesca Tasca wint Jaarprijs 2022 - Spierfonds Francesca Tasca (Marie Skłodowska-Curie Ph.D.Underpinning these investigations, we develop and integrate gene delivery and gene editing technologies grounded on recombinant viruses and programmable nucleases, e.g., adenoviral vectors and CRISPR systems, respectively.Īdditional information about our team and research activities is available via the links: This research seeks to achieve seamless and scarless chromosomal DNA editing for modeling or repairing genetic defects in pluripotent stem cells and tissue-specific stem/progenitor cells. We further study the genome editing outcomes (wanted and unwanted) resulting from using nucleases versus “nickases” and donor DNA substrates with different structures and topologies. Gene editing tools under investigation include programmable DNA-cleaving enzymes (nucleases and “nickases”) and exogenous DNA-repairing templates (donor DNA). We investigate new genome editing principles based on the activation and guiding of specific DNA repair processes after the delivery of gene editing tools into target cells. Important portions of our research builds upon the pioneering role of the Leiden University and the LUMC on adenoviral vector research, hematopoietic stem cell transplantation and Duchenne muscular dystrophy. ![]() These interconnected research lines seek to contribute to the treatment of monogenetic disorders of the striated musculature and hematopoietic system and, to this end, include collaborations with colleagues from the LUMC, other Dutch institutions and European networks. The resulting findings and toolboxes are starting to be directed towards the modeling and correction of genetic disorders. The Genome editing group at the Department of Cell and Chemical Biology of the LUMC focuses its research on these two critical aspects by investigating (i) viral vectors as delivery agents of gene-editing tools and (ii) seamless gene-editing strategies which, in contrast to conventional approaches, do not generate mutagenic double-stranded breaks at chromosomal sequences. Yet, critical improvements at the levels of gene-editing tool delivery and precision are in demand. ![]() The technology is expected to have a significant impact in enabling cell therapies to be progressed through clinical development and towards commercialization.Rapid progress in the genome editing field is contributing to solving complex scientific questions and to develop advanced gene and cell therapies for tackling genetic disorders with unmet medical needs and high societal burdens. Horizon reports that compared to currently available gene editing methodologies such as CRISPR/Cas9-which creates ‘cuts’ in the gene that can lead to adverse or negative effects-this new technology allows for more accurate gene editing while reducing unintended genomic changes. According to Horizon, the technology has potential applications in the development of new cell therapies and will expand the company’s research tools and services.īase editing is a technology platform for engineering DNA or genes in cells that has the potential to correct errors or mutations in the DNA by modifying genes using an enzyme. Horizon will also fund further research in base editing at Rutgers while undertaking evaluation and proof-of-concept studies at Horizon. As part of the agreement, Horizon has made a non-material payment to Rutgers for an option to exclusively license the base editing technology for use in all therapeutic applications. ![]() Shengkan Jin, associate professor of pharmacology at Rutgers Robert Wood Johnson Medical School. Under the partnership, Horizon and Rutgers will further develop the base editing platform from the laboratory of Dr. 28, 2019, gene editing company Horizon Discovery Group announced that it has entered into an exclusive strategic partnership with Rutgers, The State University of New Jersey, to develop and commercialize a gene editing technology known as base editing.
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