THERAPY AND PREVENTION OF TDP-43 PROTEINOPATHY

摘要

The present invention provides methods for the therapy and prevention of TDP-43 proteinopathies and for the treatment of neuronal cells having elevated cytoplasmic TDP-43 comprising administration of one or more modulators of OPRS1 activity and/or level and/or expression sufficient to reduce, inhibit, prevent or delay cytoplasmic sequestration and/or cytoplasmic accumulation of TDP-43 protein in neuroglia and/or neuronal cells. The present invention also provides methods of identifying, isolating and formulating modulators of OPRS1 activity and/or level and/or expression for use in the preparation of a compositions for the treatment or prevention of a TDP-43 proteinopathy or for reducing cytoplasmic sequestration and/or cytoplasmic accumulation of TDP-43 protein in a neuronal cell having cytoplasmic elevated TDP-43 level.