Compositions and methods for modulating rpgr expression
AssigneeUniversity of Massachusetts
InventorsHemant Khanna, Laura Moreno Leon
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Abstract
Aspects of the disclosure relate to compositions and methods for modulating expression of certain RPGR retinitis pigmentosa GTPase regulator (RPGR) proteins in a cell or subject. In some embodiments the disclosure provides expression vectors encoding one or more RPGR proteins and/or one or more inhibitory nucleic acids that target certain isoforms of RPGR, for example RPGRCONST. In some aspects, the disclosure relates to methods of treating retinitis pigmentosa (RP) in a subject by administering compositions that modulate RPGR expression or activity.
Claims33
An isolated nucleic acid comprising an expression cassette comprises a first region that encodes an inhibitory nucleic acid that specifically binds to a nucleic acid sequence encoding a RPGRCONST protein.
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An isolated nucleic acid comprising an expression construct that comprises an inhibitory nucleic acid that comprises the sequence set forth in SEQ ID NO:
- 11 or 14 and specifically binds to a nucleic acid sequence encoding a RPGRORF15 protein.
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An isolated inhibitory nucleic acid comprising a binding site for a microRNA (miRNA) which binds an RPGR messenger RNA (mRNA).
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Dependencies are inferred from the claim text.