Skin genetically engineered as a bioreactor or a 'metabolic sink'.
Genetically manipulated human keratinocytes can produce and secrete medically relevant proteins to the circulation. Genetically modified skin may also function as a 'metabolic sink' detoxifying the body of metabolites which accumulate in certain metabolic diseases. At the National Institutes of Health (NIH), Bethesda, Md., a clinical trial investigating the treatment of an ocular disease using the skin as a 'metabolic sink' for ornithine accumulating in gyrate atrophy patients is being prepared. The trial will involve the transplantation of a small patch of autologous keratinocytes, transduced ex vivo, onto the thighs of patients with gyrate atrophy. We are now investigating other diseases where this technology may be applicable such as in the treatment of hyperphenylalaninemia or hypercholesterolemia.
Christensen
Rikke
R
Institute of Human Genetics, University of Aarhus, Denmark.
Jensen
Uffe Birk
UB
Jensen
Thomas G
TG
D019149:Bioreactors; D000078404:Epidermal Cells; D004817:Epidermis / Q000378:metabolism*; D018014:Gene Transfer Techniques / Q000639:trends; D005818:Genetic Engineering / Q000379:methods* / Q000639:trends; D006801:Humans; D015603:Keratinocytes / Q000166:cytology / Q000378:metabolism* / Q000637:transplantation*; D008657:Metabolic Clearance Rate / Q000235:genetics; D008659:Metabolic Diseases / Q000628:therapy*; D013234:Stem Cells / Q000166:cytology / Q000378:metabolism; D014182:Transplantation, Autologous / Q000379:methods* / Q000639:trends