title: A knot polymer mediated non-viral gene transfection for skin cells.
abstract: A knot polymer, poly[bis(2-acryloyl)oxyethyl disulphide-co-2-(dimethylamino) ethyl methacrylate] (DSP), was synthesized, optimized and evaluated as a non-viral vector for gene transfection for skin cells, keratinocytes. With recessive dystrophic epidermolysis bullosa keratinocytes (RDEBK-TA4), the DSP exhibited high transfection efficacy with both Gaussia luciferase marker DNA and the full length COL7A1 transcript encoding the therapeutic type VII collagen protein (C7). The effective restoration of C7 in C7 null-RDEB skin cells indicates that DSP is promising for non-viral gene therapy of recessive dystrophic epidermolysis bullosa (RDEB).
authors.affiliation: Charles Institute of Dermatology, University College Dublin, Dublin 4, Ireland. dezhong.zhou@ucd.ie wenxin.wang@ucd.ie.
authors.forename: Lara
authors.initials: L
authors.lastname: Cutlar
authors.affiliation: Charles Institute of Dermatology, University College Dublin, Dublin 4, Ireland. dezhong.zhou@ucd.ie wenxin.wang@ucd.ie.
authors.forename: Yongsheng
authors.initials: Y
authors.lastname: Gao
authors.affiliation: Charles Institute of Dermatology, University College Dublin, Dublin 4, Ireland. dezhong.zhou@ucd.ie wenxin.wang@ucd.ie.
authors.forename: Ahmed
authors.initials: A
authors.lastname: Aied
authors.affiliation: Charles Institute of Dermatology, University College Dublin, Dublin 4, Ireland. dezhong.zhou@ucd.ie wenxin.wang@ucd.ie.
authors.forename: Udo
authors.initials: U
authors.lastname: Greiser
authors.affiliation: Division of Molecular Dermatology and EB House Austria, Department of Dermatology, Paracelsus Medical University, Salzburg, Austria.
authors.forename: Eva Maria
authors.initials: EM
authors.lastname: Murauer
authors.affiliation: Charles Institute of Dermatology, University College Dublin, Dublin 4, Ireland. dezhong.zhou@ucd.ie wenxin.wang@ucd.ie.
authors.forename: Dezhong
authors.initials: D
authors.lastname: Zhou
authors.affiliation: Charles Institute of Dermatology, University College Dublin, Dublin 4, Ireland. dezhong.zhou@ucd.ie wenxin.wang@ucd.ie.
authors.forename: Wenxin
authors.initials: W
authors.lastname: Wang
mesh_terms: D024143:Collagen Type VII / Q000737:chemistry* / Q000235:genetics* / Q000378:metabolism; D018076:DNA, Complementary / Q000235:genetics; D004123:Dimethylamines / Q000138:chemical synthesis* / Q000737:chemistry; D016108:Epidermolysis Bullosa Dystrophica / Q000235:genetics* / Q000378:metabolism; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000737:chemistry* / Q000378:metabolism; D006801:Humans; D008689:Methacrylates / Q000138:chemical synthesis* / Q000737:chemistry; D011108:Polymers / Q000737:chemistry* / Q000378:metabolism; D012867:Skin / Q000737:chemistry* / Q000378:metabolism; D014162:Transfection
chemical_list: D024143:Collagen Type VII; D018076:DNA, Complementary; D004123:Dimethylamines; D008689:Methacrylates; D011108:Polymers; C000613672:poly(bis(2-acryloyl)oxyethyl disulphide-co-2-(dimethylamino)ethyl methacrylate