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Genterapi til sygdomme i centralnervesystemet

Forfatter(e)
Kristoffer Nissen1, Louise Klem1, Rose Jeppesen1, Tobias Melton Axelsen2, Søren Hofmann Christiansen1, Casper Rene Gøtzsche1, Lars Pinborg3, Bo Jespersen4, Kristian Klemp5 & David Woldbye1 1) Institut for Neuroscience, Panum Instituttet, Københavns Universitet2) Klinik for Hjerne- og Nervesygdomme, Herlev Hospital3) Epilepsiklinikken & Neurobiologisk Forskningsenhed, Neurologisk Klinik, Rigshospitalet4) Neurokirurgisk Klinik, Rigshospitalet5) Øjenklinikken, Rigshospitalet

Ugeskr Læger 2020;182:V09190535
Reference: 
Ugeskr Læger 2020;182:V09190535

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.

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