Abstract: The technology described herein is directed to pharmaceutical compositions comprising at least one Scg2 neuropeptide, as well as cell culture media or kits comprising such Scg2 neuropeptides. Also described herein are nucleic acids, vectors, or viral vectors encoding at least one Scg2 neuropeptide. In further aspects, described herein are methods of treating a memory-associated disorder, a learning disability, a neurodegenerative disease or disorder, or epilepsy with a pharmaceutical composition, nucleic acid, vector, or viral vector as described herein. In further aspects, described herein are detection methods of memory-associated analytes, such as Scg2 neuropeptides.

Abstract: The present disclosure, at least in part, relates to compositions (e.g., isolated nucleic acid and rAAVs) and methods for treating Non-syndromic hearing loss and deafness (DFNB1) by delivering gap junction beta 2 (GJB2) protein to inner ear cells that normally express GJB2 (e.g., fibrocytes and supporting cells of the organ of Corti and nearby regions). The isolated nucleic acid of the present disclosure comprises an expression cassette, wherein the expression cassette comprises a gap junction beta 2 (GJB2) gene regulatory element (GRE) (e.g., GJB2 enhancers, GJB2 promoters, GJB2 5? UTR, and/or GJB2 3? UTR), and a nucleotide sequence encoding a GJB2 protein.

Abstract: The technology described herein is directed to adeno-associated vims (AAV) vectors comprising at least one gene regulatory element (GRE) and cells comprising said vectors. In another aspect, described herein are methods of screening for said gene regulatory elements. In another aspect, described herein are nucleic acid compositions comprising a GRE as described herein.

Abstract: We have determined that MeCP2 protein mediates modulation of long-gene expression in the brain and results in neurological dysfunction associated with autism spectrum disorders, including but not limited to, Fragile X Syndrome, Rett syndrome, and Angelman syndrome (AS). In particular, a lack of MeCP2 protein causes up-regulation of long gene expression in the brain which corresponds with the pathology of Rett syndrome and Fragile X Syndrome, while too much MeCP2 protein results in excessive repression of long gene expression in the brain and pathology related to MeCP2 duplication syndrome. Accordingly, embodiments of the invention are directed to methods for treatment of autism spectrum disorders. The methods involve administration, to a subject, agents that modulate the expression of long genes in the brain.

Abstract: The invention provides methods of screening a compound that can increase spine/excitatory synapse formation and/or numbers. The compound is identified by contacting Ephexin5 with a test compound and selecting the compounds that inhibit Rho GEF activity of Ephexin5. Additionally, the invention also provides methods for increasing spine/excitatory synapse formation and/or numbers by contacting a neuron with an Ephexin5 inhibitor.

Abstract: Methods for the treatment of Angelman Syndrome autism spectrum disorders are provided. The methods comprise administrating to a subject an agent that increases the expression of, or increases activity of, ?-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor (AMPAR) at neuronal synapses.

Abstract: The invention provides methods of screening a compound that can increase spine/excitatory synapse formation and/or numbers. The compound is identified by contacting Ephexin5 with a test compound and selecting the compounds that inhibit Rho GEF activity of Ephexin5. Additionally, the invention also provides methods for increasing spine/excitatory synapse formation and/or numbers by contacting a neuron with an Ephexin5 inhibitor.

Abstract: The invention provides methods of screening a compound that can increase spine/excitatory synapse formation and/or numbers. The compound is identified by contacting Ephexin5 with a test compound and selecting the compounds that inhibit Rho GEF activity of Ephexin5. Additionally, the invention also provides methods for increasing spine/excitatory synapse formation and/or numbers by contacting a neuron with an Ephexin5 inhibitor.

Abstract: The invention provides methods of screening a compound that can increase spine/excitatory synapse formation and/or numbers. The compound is identified by contacting Ephexin5 with a test compound and selecting the compounds that inhibit Rho GEF activity of Ephexin5. Additionally, the invention also provides methods for increasing spine/excitatory synapse formation and/or numbers by contacting a neuron with an Ephexin5 inhibitor.

Abstract: Methods for the treatment of Angelman Syndrome autism spectrum disorders are provided. The methods comprise administrating to a subject an agent that increases the expression of or increases activity of, ?-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor (AMPAR) at neuronal synapses.