Strategies for long-term gene expression in the skin to treat metabolic disorders

Abstract

Due to its accessibility, size and contact with the blood circulation, the skin is an attractive target for somatic gene therapy. Permanent cutaneous expression can be achieved by genetic manipulation of epidermal keratinocytes ex vivo followed by transplantation or by local injection of viral vectors. Furthermore, progress is being made to develop topical gene transfer methods leading to permanent gene expression. There is experimental evidence showing that genetically engineered skin can produce and secrete medically relevant proteins to the circulation and also produce enzymes that can clear metabolites accumulating in various diseases. Thus, cutaneous gene transfer approaches may be relevant not only for local skin diseases, but also for certain systemic disorders.

• bioreactor
• gene therapy
• metabolic diseases
• metabolic sink
• vectors