Abstract: Provided herein are improved expression constructs for the expression of ATP7B, vectors, and pharmaceutical compositions comprising such constructs. Also provided are methods of treating disease, including, but not limited to, Wilson's Disease.

Abstract: The application relates to nucleic acid regulatory elements (NAREs) that are able to enhance expression of genes in the liver. The application further relates to methods employing NAREs and uses of the NAREs. Expression cassettes and vectors containing NAREs are also disclosed. These are particularly useful for applications using gene therapy.

Abstract: This disclosure provides BDNF expression constructs and vectors comprising such constructs for treating metabolic disorders, including, but not limited to, obesity. The disclosed BDNF gene therapy is more effective than existing BDNF-based gene therapy constructs.

Abstract: The application relates to nucleic acid regulatory elements that are able to enhance expression of genes constitutively in a variety of tissues, or in particular tissues including liver, muscle, and the CNS. The application further relates to methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. These nucleic acid regulatory elements are particularly useful for applications using gene therapy.

Abstract: The present disclosure relates to a method of inducing a neuroprotective state comprising administering a Bromodomain and Extra-Terminal motif (BET) inhibitor under conditions effective to induce a neuroprotective state. Also disclosed are methods of preventing and/or treating neurodegenerative disease, methods of reducing microglial inflammation, and methods of restoring microglial homeostasis, where the methods include administering a Bromodomain and Extra-Terminal motif (BET) inhibitor.