Genterapi til sygdomme i centralnervesystemet
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Genterapi til sygdomme i centralnervesystemet. / Nissen, Kristoffer; Klem, Louise; Jeppesen, Rose; Axelsen, Tobias Melton; Christiansen, Søren Hofmann; Gøtzsche, Casper Rene; Pinborg, Lars; Jespersen, Bo; Klemp, Kristian; Woldbye, David.
In: Ugeskrift for Laeger, Vol. 182, No. 3, V09190535, 2020.Research output: Contribution to journal › Journal article › Research › peer-review
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TY - JOUR
T1 - Genterapi til sygdomme i centralnervesystemet
AU - Nissen, Kristoffer
AU - Klem, Louise
AU - Jeppesen, Rose
AU - Axelsen, Tobias Melton
AU - Christiansen, Søren Hofmann
AU - Gøtzsche, Casper Rene
AU - Pinborg, Lars
AU - Jespersen, Bo
AU - Klemp, Kristian
AU - Woldbye, David
PY - 2020
Y1 - 2020
N2 - 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.
AB - 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.
M3 - Tidsskriftartikel
C2 - 32052730
VL - 182
JO - Ugeskrift for Laeger
JF - Ugeskrift for Laeger
SN - 0041-5782
IS - 3
M1 - V09190535
ER -
ID: 260117127