Methods and compositions for treatment of ocular disorders and blinding diseases
AssigneeThe Trustees of the University of Pennsylvania
InventorsJeannette Bennicelli, Jean Bennett, Junwei Sun
Life of this patent
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AssigneeThe Trustees of the University of Pennsylvania
InventorsJeannette Bennicelli, Jean Bennett, Junwei Sun
Estimating the term…
Codon optimized nucleic acid sequences for the long form and short form of RdCVF are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequences. Recombinant vectors are provided that express the codon optimized RdCVFL and RdCVF individually, or express two copies of a codon optimized RdCVF or RdCVFL nucleic acid sequence, or both RdCVFL and RdCVF in a single vector or virus. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence or inappropriate expression of RdCVF and RdCVFL.
An expression cassette comprising one or more of:
An rAAV vector comprising AAV inverted terminal repeat sequences and an expression cassette comprising at least one of the nucleic acid sequences set forth in nucleotides 20 to 655 of SEQ ID NO:
A composition comprising a nucleic acid molecule useful in the treatment of an ocular disease comprising:
Dependencies are inferred from the claim text.