Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
5522770 | Stem Cell Research | 2016 | 6 Pages |
Abstract
Peripheral blood was collected from a 39-year-old unaffected female carrier of an X-linked recessive mutation of Iduronate 2-sulfatase gene (NM_000202.7(IDS):c.85CÂ >Â T) causing MPS II (OMIM 309900). Peripheral blood mononuclear cells (PBMCs) were reprogrammed by lentiviral delivery of a self-silencing hOKSM polycistronic vector. The pluripotency of iPSC line was confirmed by the expression of pluripotency-associated markers and in vitro spontaneous differentiation towards the 3 germ layers. The iPSC showed normal karyotype. The line offers a good platform to study MPS II pathophysiology, for drug testing, early biomarker discovery and gene therapy studies.
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Authors
Eszter Varga, Csilla Nemes, Eszter Kovács, István Bock, Norbert Varga, Anita Fehér, András Dinnyés, Julianna Kobolák,