Abstract: The present invention includes methods and compositions useful for the treatment of Dent's disease in a subject in need thereof. The invention of the present disclosure also includes a mouse model useful for the study of Dent's disease.

Abstract: Provided herein are compositions and methods for making extracellular vesicles. In some embodiments, the extracellular vesicles are used in methods for modifying cells and in methods for treating a disease in a subject.

Abstract: Provided herein are compositions and methods for editing the genome of a eukaryotic cell.

Abstract: Provided are compositions, systems, and methods useful for effecting gene editing in eukaryotic cells. Compositions include plasmids that encode one or more viral fusion proteins in which one or more viral proteins are fused with an aptamer-binding protein. Compositions also include plasmids that encode a non-viral nucleic acid sequence, wherein the non-viral nucleic acid sequence encodes a CRISPR system component. In some instances, the non-viral nucleic acid sequence also includes an aptamer sequence. The plasmids can be used to generate viral particles, including lentivirus-like particles that contain a viral fusion protein and a non-viral RNA sequence. Systems of producing such viral particles are provided. Also provided are methods of using the viral particles of the disclosure to effect gene editing in eukaryotic cells.

Abstract: The invention includes compositions and methods for the selective expression of a target gene in a subset of cells. In certain embodiments, the present invention includes a construct comprising a first nucleic acid sequence comprising an episomal maintenance element and a second nucleic acid sequence comprising a target gene wherein the expression of the episomal maintenance element is regulated by a constitutive promoter and the expression of the target gene is regulated by a non-constitutive promoter. The construct is able to maintain episomal state, no matter whether the target gene is expressed in the cell.

Abstract: The invention includes compositions and methods for the selective expression of a target gene in a subset of cells. In certain embodiments, the present invention includes a construct comprising a first nucleic acid sequence comprising an episomal maintenance element and a second nucleic acid sequence comprising a target gene wherein the expression of the episomal maintenance element is regulated by a constitutive promoter and the expression of the target gene is regulated by a non-constitutive promoter. The construct is able to maintain episomal state, no matter whether the target gene is expressed in the cell.

Abstract: MEX3C deficiency impairs the development of white and brown adipose tissue. Hence the present invention provides, among other things, a method of screening a candidate compound for activity in inhibiting fat deposition in a subject in need thereof and/or treating a condition in a subject in need thereof, comprising: (a) contacting a candidate compound to a cell that expresses MEX3C protein; and then (b) detecting a quantity of expression of the MEX3C protein in the cell; a depression in the expression of MEX3C protein when the candidate compound is contacted thereto as compared to that expressed when the candidate compound is not contacted thereto indicating the compound is active in inhibiting fat deposition and/or treating a condition in a subject in need thereof. Methods of treatment and screening subjects are also described.

Abstract: MEX3C deficiency impairs the development of white and brown adipose tissue. Hence the present invention provides, among other things, a method of screening a candidate compound for activity in inhibiting fat deposition in a subject in need thereof and/or treating a condition in a subject in need thereof, comprising: (a) contacting a candidate compound to a cell that expresses MEX3C protein; and then (b) detecting a quantity of expression of the MEX3C protein in the cell; a depression in the expression of MEX3C protein when the candidate compound is contacted thereto as compared to that expressed when the candidate compound is not contacted thereto indicating the compound is active in inhibiting fat deposition and/or treating a condition in a subject in need thereof. Methods of treatment and screening subjects are also described.