Scientists discover new approach to gene therapy
Hubrecht InstitutePeer-Reviewed Publication
Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate piece of DNA was cut out using CRISPR-Cas9 technology. This strategy opens up new possibilities for treating genetic diseases. The team specifically shows the technology’s potential for the treatment of sickle cell disease and beta-thalassemia, two genetic blood diseases. In these conditions, a faulty gene could potentially be compensated by reactivating a helpful but normally inactive one. This ‘delete-to-recruit’ method works by simply changing the spacing—without adding new genes or foreign elements. The discovery, made by researchers from the Hubrecht Institute (De Laat group), Erasmus MC and Sanquin, was published in the journal Blood.
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- USEQ is subsidized by the University Medical Center Utrecht, USEQ is subsidized by The Netherlands X-omics Initiative, Research in the laboratory of WdL was financially supported by the EU Horizon 2020-funded Innovative Training Network ‘Molecular Basis of Human Enhanceropathies’ (Enhpathy,www.enhpathy.eu), under Marie Sklodowska-Curie grant, Research in the laboratory of WdL was financially supported by an NWO Groot grant from the Netherlands Organisation for Scientific Research (NWO), Research in the laboratory of WdL was financially supported by Oncode Institute Base Funding, Work in the laboratory of SP was supported by TKI Health Holland, Work in the laboratory of SP was supported by ZonMw PSIDER consortium TRACER, Work in the laboratory of SP was supported by EU Horizon Europe Pathfinder EdiGenT, Work in the laboratory of SP was supported by NWO Applied and Engineering Sciences Open Technology Programme, Work by the laboratory of EvdA was supported by Sanquin Blood Supply grant, Work by the laboratory of EvdA was supported by Sanquin research fund