Means and methods to treat dystonia
AssigneesVib Vzw, Katholieke Universiteit Leuven, KU LEUVEN R&D
InventorsRose Goodchild, Micheline Grillet
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Abstract
The present application relates to the field of neurological diseases, particularly to dystonia, even more particularly to primary dystonia, most particularly DYT1 primary dystonia. It is disclosed that the DYT1 dystonia causative mutation in TORSIN1A leads to hyperactivation of LIPIN. The invention provides substances modulating LIPIN function, in particular RNA molecules inhibiting LIPIN function and medical uses of these LIPIN inhibitors. Methods are disclosed to screen for medicaments that counteract the effects of TORSIN1A mutation.
Claims3
A method for the treatment of a neurological disease in a subject, the method comprising:
- administering to the subject a nucleic acid sequence that is complementary to a nucleic acid sequence encoding Lipin1 thereby inhibiting the functional expression of LIPIN1 in the subject;
- wherein the neurological disease is dystonia caused by at least one mutation in the TORSIN1A gene.
A method of reducing the functional expression of LIPIN1 in a subject, the method comprising:
- administering to the subject a nucleic acid sequence that is complementary to a nucleic acid sequence encoding Lipin1 that can reduce expression of Lipin1;
- wherein the subject has at least one mutation in the TORSINA gene.
Dependencies are inferred from the claim text.