COMPOUNDS AND METHODS OF TREATING USHER SYNDROME III

摘要

An isolated polynucleotide includes a nucleic acid sequence that includes a cDNA coding sequence of a clarin-1 gene and a 3′UTR nucleic acid that is derived from the 3′UTR of the clarin-1 gene. The 3′UTR nucleic acid can enhance expression of clarin-1 in a cell transfected with the polynucleotide compared to a cell transfected with a similar polynucleotide devoid of the 3′UTR nucleic acid.