Abstract: Provided herein are compositions and methods related to treating repeat expansion diseases that feature the sequestration of Muscleblind-like (MBNL) proteins by the toxic repeat RNA transcripts within distinct nuclear foci that are expressed from the expanded repeat tracts. Certain compositions of synthetic MBLN proteins can be used to displace endogenous MBNL from the toxic RNAs or replace endogenous MBNL within the cell for normal function.

Abstract: Provided herein are compositions and methods related to treating repeat expansion diseases that feature the sequestration of Muscleblind-like (MBNL) proteins by the toxic repeat RNA transcripts within distinct nuclear foci that are expressed from the expanded repeat tracts. Certain compositions of synthetic MBLN proteins can be used to displace endogenous MBNL from the toxic RNAs or replace endogenous MBNL within the cell for normal function.

Abstract: A specialized drug delivery unit for inner ear treatment which employs a portion of carrier media material containing one or more therapeutic agents therein. The carrier media material is designed to release the therapeutic agents in a controlled manner over time. The drug delivery unit is sized for placement in the round window niche of a patient. The released therapeutic agents come in contact with the round window membrane and pass therethrough into the inner ear for treatment purposes. This system provides many benefits ranging from the ability to deliver drugs in a site specific, highly controlled manner to the transfer of such materials with minimal patient discomfort and monitoring requirements.

Abstract: Micro-reactors for selective enhancement of ligands by exponential enrichment (ISOS) include a reactant chamber defined in a fluoropolymer. A target exposure aperture controls a surface area, crystal face or orientation and surface features of a target that is exposed to a random mixture of candidate molecules. The surface area of the target can be selected based on number or concentration of candidate species to enhance candidate competition. The target surface can be formed by deposition of a thin film of a target material on a rigid substrate such as a glass plate. Selected exposure areas are typically substantially smaller than a characteristic cross sectional area of a reactant chamber volume and can be at least as small as 0.1 mm2.

Abstract: The present invention provides methods and devices for controlled delivery of a therapeutic agent to an internal cavity of the ear, particularly to the inner ear. In general, the invention uses a drug delivery unit for inner ear treatment which employs a portion of carrier media material containing one or more therapeutic agents therein. The carrier media material is designed to release the therapeutic agents in a controlled manner over time. The drug delivery unit is shaped and sized for placement of at least a portion thereof in the round window niche of a patient. The released therapeutic agents come in contact with the round window membrane and pass therethrough into the inner ear for treatment purposes. This system provides many benefits ranging from the ability to deliver drugs in a site specific, highly controlled manner to the transfer of such materials with minimal patient discomfort and monitoring requirements.