The need for regulatable vectors for gene therapy for Parkinson's disease.
Gene therapy is now a very promising approach for the treatment of Parkinson's disease, for which there are currently few treatment options. However, gene therapy is invasive and irreversible, and its long-term effects are not yet known. Regulatable vectors allow the expression of the introduced gene to be adjusted or stopped by changing the dose of an oral inducer drug, thus adding an important safety mechanism as well as the ability to tailor the dose to an individual patient's needs. Although the use of conventional gene therapy should not be delayed until regulatable systems are available, clinical trials of regulatable gene therapies are imminent. Regulatable systems provide the best hope for safely delivering effective, flexible treatments over the long course of Parkinson's disease, and their development should be actively supported.
Cress
Dean E
DE
Intrexon Corporation, Blacksburg, VA 24060, USA. dcress@intrexon.com
D000818:Animals; D004298:Dopamine / Q000096:biosynthesis / Q000235:genetics; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D010300:Parkinson Disease / Q000235:genetics / Q000628:therapy*
D004298:Dopamine