Why is the international profile of partners an asset to the project ?

Genome editing technologies based on CRISPR/Cas systems allow targeted genomic modification with unprecedented precision and have emerged as powerful alternatives to the conventional gene therapy approaches for various human diseases, with a series of clinical trials in progress.

However, some crucial challenges remain to be addressed to enhance efficiency and safety and decrease costs of treatments. Current viral-based delivery systems are associated with high risk of toxicity and immunogenicity and remain highly expensive.

ERASMUS MC

Erasmus University Medical Center Rotterdam (EMC) is committed to a healthy population and excellence in healthcare through research and education.

EMC has been awarded the “HR Excellence in Research” by the European Commission. This recognizes institutions that perform good practices in recruitment and hosting of researchers. EMC has broad expertise in various research fields, ranging from fundamental and clinical domains to public health and prevention.

Bibliometric indicators place EMC in the top 20 of clinical medicine worldwide. In addition to scientific research, patient care and education are core tasks of EMC. It is the top referral center for a region of about five million inhabitants and the largest medical school in The Netherlands, with around 3,500 medical students and 250 PhD graduates annually.

The EMC Department of Cell Biology has over 100 scientific staff; the Philipsen group focusses on molecular control of erythropoiesis. www.erasmusmc.nl

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Sjaak Philipsen obtained his PhD in 1988 from the State University of Groningen (NL). There, he studied estrogen-dependent induction of chicken yolk protein genes in the liver.

He was awarded an EMBO long-term fellowship and joined the National Institute for Medical Research (London, UK) to functionally dissect the beta-globin Locus Control Region.

In 1993 he moved to Erasmus MC (Rotterdam, NL) to study developmental regulation of the beta-like globin genes.

Recently, he expanded these investigations to include gene editing approaches in human erythroid cells, with the aim to develop novel therapies for beta-thalassemia and sickle cell disease patients.