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The Geijsen group studies human genetic disorders, such as muscular dystrophy using gene editing techniques.
Pluripotent stem cells can divide almost indefinitely and have the unique ability to give rise to every cell type in our body. For this reason, they are the most versatile source of stem cells for (drug) research and transplantation therapy. The first human pluripotent stem cell lines were derived from early un-implanted blastocyst embryos, that develop about 6-8 days after fertilization. Such embryos are typically left over after in vitro fertilization (IVF) treatment and would otherwise have remained frozen or be discarded. While human embryonic stem cells have the potential to revolutionize the ways we study and treat human disease, their moral status has also been the source of intense ethical debate.
Recently, a novel method was developed that allows the generation of pluripotent stem cells from mature cell types such as skin cells. These so-called induced pluripotent stem cells (iPS cells) are very similar to embryonic stem cells, and have the same capacity of forming all the different cell types in our body. Yet, because no embryo is destroyed in the process of creating iPS cells, these cells are free from the ethical dilemmas associated with embryonic stem cells.
The process of creating iPS cells, often referred to as “reprogramming,” involves introducing a combination of three to four genes into a mature cell. Under influence of these reprogramming factors, the mature (skin) cell converts back into an embryonic stem cell-like state. Since iPS cells can also be derived from patient samples, they give us unique new ways to study human disease in a petridish.
The Geijsen group is developing novel iPS-based methods to study human development and disease and to develop stem cell-based assays for drug-screening, pathway identification and toxicology assessments.
Chen HH, Welling M, Bloch DB, Muñoz J, Mientjes E, Chen X, Tramp C, Wu J, Yabuuchi A, Chou YF, Buecker C, Krainer A, Willemsen R, Heck AJ, Geijsen N.
West JA, Park IH, Daley GQ, Geijsen N.
Geijsen N, Horoschak M, Kim K, Gribnau J, Eggan K, Daley GQ.
Buecker C, Chen HH, Polo JM, Daheron L, Bu L, Barakat TS, Okwieka P, Porter A, Gribnau J, Hochedlinger K, Geijsen N.
Chou Y-F, Chen H-H, Eijpe M, Yabuuchi A, Chenoweth JG, Tesar P, Lu J, McKay RDG, Geijsen N.
Niels Geijsen is group leader at the Hubrecht Institute professor of Regenerative Medicine at Utrecht University. His group focuses on understanding the biology of pluripotent stem cells and developing new stem-cell based tools to explore human development and oncogenic transformation. In addition, the Geijsen group uses the CRISPR/Cas technology to study mutations that cause diseases such as FSHD (Facioscapulohumeral muscular dystrophy). Recently they have developed a new technology, called iTOP, to deliver the molecules needed for CRISPR/Cas into cells.
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