Kristoffer Nissen, Louise Klem, Rose Jeppesen, Tobias Melton Axelsen, Søren Hofmann Christiansen, Casper Rene Gøtzsche, Lars Pinborg, Bo Jespersen, Kristian Klemp & David Woldbye:
Gene therapy for central nervous system disorders
Ugeskr Læger 2020;182:V09190535
In recent years, gene therapy has resurged as a potential treatment for an increasing number of medical diseases including those affecting the central nervous system (CNS), which is discussed in this review. Clinical trials have revealed promising results particularly in gene therapy for Parkinson’s disease with upregulation of dopamine synthesis or downregulation of huntingtin synthesis in Huntington’s disease. Gene therapy for spinal motor atrophy has received FDA approval this year. The biggest success is seen in ophthalmology, where gene therapy has been FDA/EU-approved for retinitis pigmentosa, sparking further hope of use for other CNS diseases in a near future.