Agent for treating or preventing a dominantly-inherited disease
AssigneeThe University of Adelaide
InventorsPaul Quinton THOMAS, Fatwa ADIKUSUMA, Ashleigh Bryar GEIGER
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Abstract
An agent for treating a common form of autosomal dominant retinitis pigmentosa (ADRP) is disclosed, wherein the agent comprises a first nucleotide sequence encoding a CRISPR-associated (Cas) endonuclease which binds to an NG or NNGRRT PAM (protospacer adjacent motif) sequence, and a second nucleotide sequence encoding or comprising a guide RNA (gRNA) capable of forming a CRISPR-Cas complex with said Cas endonuclease, wherein the gRNA is specifically targeted to a target mutant allele selected from RHOP23H and NR2E3G56R.
Claims16
-26. (canceled)
An agent for treating a common form of autosomal dominant retinitis pigmentosa (ADRP) in a subject, wherein said agent comprises:
- (i) a first nucleotide sequence encoding a CRISPR-associated (Cas) endonuclease which binds to an NG protospacer adjacent motif (PAM) sequence;
- and
- (ii) a second nucleotide sequence encoding or comprising a guide RNA (gRNA) capable of forming a CRISPR-Cas complex with said Cas endonuclease, wherein said gRNA is specifically targeted to:
- a RHOP23H target mutant allele and comprises a targeting nucleotide sequence selected from the group consisting of SEQ ID NOs:3, 6 and 8;
- or
- a NR2E3G56R target mutant allele and comprises a targeting nucleotide sequence selected from the group consisting of SEQ ID NOs:17 and 18.
A CRISPR-Cas complex comprising a CRISPR-associated (Cas) endonuclease which binds to an NG PAM sequence and a gRNA, wherein said gRNA is specifically targeted to:
- a RHOP23H target mutant allele and comprises a targeting nucleotide sequence selected from the group consisting of SEQ ID NOs:3, 6 and 8;
- or
- a NR2E3G56R target mutant allele and comprises a targeting nucleotide sequence selected from the group consisting of SEQ ID NOs:17 and 18.
Dependencies are inferred from the claim text.