Abstract: Disclosed is a method of preventing, treating or reducing a lysosomal storage disorder (LSD) or a symptom thereof in a subject in need thereof comprising administering a therapeutically effective amount of caspase inhibitor to the subject. The therapeutically effective amount of caspase inhibitor may be a medicament or a composition for preventing, treating or reducing a lysosomal storage disorder (LSD) or a symptom thereof in a subject comprising a caspase inhibitor, and optionally a pharmaceutically acceptable carrier. One preferred caspase inhibitor is the pan caspase inhibitor Emricasan.

Abstract: Disclosed is a rapid, non-invasive and highly specific and sensitive diagnostic assay for the identification of individuals with synucleinopathy and for measuring synucleinopathy progression or status. Test kits for diagnosis of an individual suspected of having synucleinopathy are also disclosed.

Abstract: Disclosed herein is a small peptide, LaR2C, corresponding to the C terminus of RRM2 of the human La protein that binds to the IRES element of hepatitis C virus RNA and its derivatives. This invention demonstrates that human La protein interacts with the HCV IRES element both in vitro and in vivo and also shown that this interaction enhances the efficiency of viral RNA translation (Pudi et al, J of Biol Chem, 2003). La protein has three putative RNA recognition motifs (RRM1-3). It has been established that RRM2 binds with high affinity around the GCAC sequence near the initiator AUG and the binding induces a conformational change in the HCV IRES which is critical for the internal initiation of translation (Pudi et al, J of Biol Chem, 2004).

Abstract: Disclosed herein is a small 7 amino-acid peptide, corresponding to the C terminus of RRM2 of the human La protein that binds to the IRES element of hepatitis C virus RNA and its derivatives. This disclosure demonstrates that this 7-mer interacts with the HCV IRES element both in vitro and in vivo and can compete against cellular La protein in binding to the HCV RNA. It is also shown here that this 7-mer peptide is able to inhibit HCV-IRES mediated translation in vivo which, in turn, leads to decreased viral replication.