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US 2023/0054569 A1Published application
Compositions and methods for treating retinitis pigmentosa
AssigneeAlia Therapeutics Srl
InventorsAntonio Casini, Laura Pezzè, Kalina Badowska
Life of this patent
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Abstract
Compositions and methods useful for altering a P347 mutation in a RHO gene.
Claims60
A guide RNA molecule (gRNA) for editing a human RHO gene having a P347 mutation.
A guide RNA molecule (gRNA) comprising a spacer whose nucleic acid sequence comprises 15 or more consecutive nucleotides from a reference sequence or comprises a nucleotide sequence that is at least 85% identical to a reference sequence, wherein the reference sequence is:
- (SEQ ID NO:
- 5)
- a) GGUCUUAGGCCAGGGCCACC;
- (SEQ ID NO:
- 6)
- b) GCCCUGGCCUAAGACCUGCCU;
- (SEQ ID NO:
- 7)
- c) CCUAGGCAGGUCUUAGGCCA;
- (SEQ ID NO:
- 8)
- d) gCCUAGGCAGGUCUUAGGCCA;
- (SEQ ID NO:
- 9)
- e) GACGAGCCAGGUGGCCCUGGCCU;
- (SEQ ID NO:
- 10)
- f) GUCCUAGGCAGGUCUUAGGCCAGG;
- (SEQ ID NO:
- 11)
- g) UUAGGCCAGGGCCACCUGGCUC;
- or
- (SEQ ID NO:
- 12)
- h) GCCAGGUGGCCCUGGCCUAAGA.
22 more elements
Dependencies are inferred from the claim text.