Genterapi til sygdomme i centralnervesystemet

Research output: Contribution to journalJournal articleResearchpeer-review

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.

Translated title of the contributionGene therapy for central nervous system disorders
Original languageDanish
Article numberV09190535
JournalUgeskrift for Laeger
Issue number3
Number of pages6
Publication statusPublished - 2020

ID: 260117127