Engrailed protein for use in the treatment of tdp-43 proteinopathies
AssigneesBrainever, Institut National de la Sante et de la Recherche Medicale, Centre National De La Recherche Scientifique, College De France
InventorsStéphanie Vargas Abonce, Alain Prochiantz
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Abstract
The present invention relates to Engrailed protein, a nucleic acid encoding Engrailed protein and compositions comprising the same for use in treating or preventing diseases associated with accumulation of TDP-43 in the cell, particularly in the cell cytoplasm. Also provided herein are in vitro methods of selecting a subject for such therapy by detecting cellular accumulation of TDP-43, particularly cellular cytoplasmic accumulation, in a biological sample of the subject. Also provided herein are in vitro methods of following a subject treated with such therapy by measuring cellular accumulation, particularly cellular cytoplasmic accumulation, of TDP-43 in a biological sample of the treated subject.
Claims11
A method for reducing cytoplasmic TDP-43 aggregates levels in cells of a subject suffering from a disease associated with cytoplasmic mislocalization and aggregation of TDP-43, by means of administration, to a patient in need thereof, of an effective amount of Engrailed protein, a nucleic acid encoding Engrailed protein or a composition comprising the Engrailed protein or the nucleic acid encoding Engrailed protein.
A method comprising a step of determining the amount of TDP-43 in cells in a biological sample obtained from a subject to determine if an increased cytoplasmic accumulation of TDP-43 is observed, wherein if an increased cytoplasmic accumulation of TDP-43, is observed, the subject can be selected for treatment with a composition comprising Engrailed protein or a nucleic acid encoding Engrailed protein.
A method of selecting a subject for treatment with a composition comprising Engrailed protein or a nucleic acid encoding Engrailed protein comprising determining the amount of TDP-43 in the cytoplasm of cells from a biological sample, particularly the amount of TDP-43 aggregates in the cytoplasm of cells, and selecting the subject for said treatment.
Dependencies are inferred from the claim text.