Genetic suppression and replacement
AssigneesElizabeth, The Provost, Fellows and Scholars of the College of the Holy and Undivided Trinity of Queen
InventorsGwyneth Jane Farrar, Sophia Millington-Ward, Naomi Chadderton, Arpad Palfi, Mary O'Reilly, Paul Kenna, Peter Humphries
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Abstract
The invention relates to gene suppression and replacement. In particular, the invention relates to enhanced expression of suppression agents for suppressing gene expression in a cell and in vivo and replacement nucleic acids that are not inhibited by the suppression agent. Regulatory elements are included in expression vectors to optimize expression of the suppression agent and/or replacement nucleic acid.
Claims23
A vector for expression of a suppression agent for a disease causing gene and/or a replacement nucleic acid that is not recognized by the suppression agent, wherein the vector comprises at least one of the conserved regions selected from:
- conserved region B from the rhodopsin gene represented by SEQ ID NO:
- 93, or a variant or equivalent thereof;
- conserved region C from the rhodopsin gene represented by SEQ ID NO:
- 94, or a variant or equivalent thereof;
- conserved region F and G from the rhodopsin gene represented by SEQ ID NO:
- 97;
- and conserved region A from the rhodopsin gene represented by SEQ ID NO:
- 92.
A method of treating ocular disease, the method comprising the step of administering the replacement nucleic acid according to 13.
Dependencies are inferred from the claim text.