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Ugeskr Laeger ; 182(3)2020 01 13.
Article in Danish | MEDLINE | ID: mdl-32052730

ABSTRACT

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.


Subject(s)
Central Nervous System Diseases , Huntington Disease , Parkinson Disease , Central Nervous System , Central Nervous System Diseases/genetics , Central Nervous System Diseases/therapy , Genetic Therapy , Humans , Parkinson Disease/genetics , Parkinson Disease/therapy
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