Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.

Provided herein are adeno-associated viruses and methods for using same to treat or prevent disorders that affect the inner ear of a subject.

Provided herein are adeno-associated viruses and methods for using same to treat or prevent disorders that affect the inner ear of a subject.

Provided herein are 2-amino-pyrazolyl-[1,2,4]triazolo [1,5a]pyridine derivatives. Such compounds are useful, for example, for the treatment and/or prevention of neurodegenerative diseases or disorders such as ophthalmological neurodegenerative disorders. This disclosure also features compositions containing the same as well as methods of using and making the same.

The present invention relates to a medicament for treating or preventing a disease related to orexin receptor, especially orexin type 2 receptor, comprising a new compound having a urea structure or a pharmaceutically acceptable salt thereof as an active ingredient. In more detail, the present invention relates to a medicament for treating or preventing narcolepsy, idiopathic hypersomnia, hypersomnia, sleep apnea syndrome, etc.

The disclosure provides a dual-vector intein-mediated protein trans-splicing system, cells, compositions, and methods of using the same for gene therapy. In some embodiments, the disclosure provides methods and compositions for treating an autosomal recessive type of non-syndromic deafness, DFNB16, by delivering a STRC gene, encoding a STRC protein, using the dual-vector system described herein.

Methods of treating a cancer or a tumor in a subject using a composition are provided. The composition includes a therapeutically effective amount of 2,4-disulfonyl a-phenyl tert-butyl nitrone (2,4-ds-PBN) or pharmaceutically acceptable salts thereof, wherein the therapeutically effective amount decreases a growth of the cancer or the tumor.

The invention provides compositions and methods for treating MPS I.

The present invention relates to a composition comprising a bilberry extract as an active ingredient for preventing or treating sensorineural hearing impairment. The bilberry extract was found to exhibit effects of inhibiting the apoptosis of auditory cells in which sensorineural damage has been induced by neomycin and increasing a reduced number of hair cells in juvenile zebra fishes such that the composition comprising the bilberry extract as an active ingredient can be provided as a pharmaceutical composition and a health food for preventing or treating various hearing impairments induced by sensorineural damage.

The present invention relates to a composition comprising a bilberry extract as an active ingredient for preventing or treating sensorineural hearing impairment. The bilberry extract was found to exhibit effects of inhibiting the apoptosis of auditory cells in which sensorineural damage has been induced by neomycin and increasing a reduced number of hair cells in juvenile zebra fishes such that the composition comprising the bilberry extract as an active ingredient can be provided as a pharmaceutical composition and a health food for preventing or treating various hearing impairments induced by sensorineural damage.