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).
Publication types
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Research Support, Non-U.S. Gov't
MeSH terms
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Collagen Type VII / chemistry*
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Collagen Type VII / genetics*
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Collagen Type VII / metabolism
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DNA, Complementary / genetics
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Dimethylamines / chemical synthesis*
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Dimethylamines / chemistry
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Epidermolysis Bullosa Dystrophica / genetics*
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Epidermolysis Bullosa Dystrophica / metabolism
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Gene Transfer Techniques
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Genetic Therapy / methods*
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Genetic Vectors / chemistry*
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Genetic Vectors / metabolism
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Humans
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Methacrylates / chemical synthesis*
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Methacrylates / chemistry
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Polymers / chemistry*
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Polymers / metabolism
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Skin / chemistry*
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Skin / metabolism
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Transfection
Substances
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Collagen Type VII
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DNA, Complementary
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Dimethylamines
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Methacrylates
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Polymers
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poly(bis(2-acryloyl)oxyethyl disulphide-co-2-(dimethylamino)ethyl methacrylate