Compositions and methods of treating usher syndrome iii
OwnerKumar N. Alagramaminventor
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Abstract
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.
Claims23
A polynucleotide comprising a nucleic acid sequence that includes a wild-type clarin-1_cDNA coding sequence having a 5′ end and a 3′ end that is flanked at the 5′ end with a 5′UTR nucleic acid sequence that is derived from the 5′UTR of the clarin-1 gene and a 3′UTR nucleic acid sequence that is derived from the 3′UTR of the clarin-1 gene, wherein the 5′UTR nucleic acid sequence and the 3′UTR nucleic acid enhance expression of clarin-1 in a cell transfected with the polynucleotide compared to a cell transfected with a similar polynucleotide devoid of the 5′UTR nucleic acid sequence and the 3′UTR nucleic acid sequence.
2-4. (canceled)
15-25. (canceled)
A vector for transfecting a cell comprising:
- a wild-type clarin-1 cDNA coding sequence having a 5′ end and a 3′ end that is flanked at the 5′ end with full-length 5′UTR nucleic acid sequence of the clarin-1 gene and at the 3′ end with full-length 3′UTR nucleic acid sequence of the clarin-1 gene.
Dependencies are inferred from the claim text.