Abstract: Compositions for increasing the expression of ACMSD in a target cell and methods of using the compositions to provide a general protective effect in a cell or tissue in a subject, or protect a subject from or treating a disease condition associated with inflammation, oxidative stress, protein aggregation, energy failure, toxic exposure such as exposure to pollutants, or any combination thereof.

Abstract: Compositions and methods for modifying the expression of one or more chitinase proteins in a target cell or tissue type are disclosed. The compositions can be used in methods of treating a disease condition associated with expression of a chitinase protein in a cell or tissue type in a subject in need thereof, such as ALS and PD.

Abstract: Engineered adeno-associated virus (AAV) capsid proteins, each having tropism to a desired target cell or tissue type, are disclosed. Also disclosed are methods of generating the engineered proteins, libraries comprising the engineered proteins, recombinant viruses comprising the engineered proteins, and nucleic acid constructs encoding the engineered proteins.

Abstract: Disclosed are systems and compositions for reducing the expression of a CaV1.3 protein in a subject and methods of using the systems for treating dyskinesias induced by DA agonist therapy including levodopa-induced dyskinesias (LID), improving the response to levodopa, and improving the response to levodopa in a subject in need thereof; and slowing progression of Parkinson's disease by providing protection against death or dysfunction of substantia nigra dopamine neurons.