Materials and methods for treatment of usher syndrome type 2A

A gRNA or sgRNA comprising a nucleotide sequence set forth in SEQ ID NO:

• 5321, SEQ ID NO:
• 5323, SEQ ID NO:
• 5325, SEQ ID NO:
• 5327, or SEQ ID NO:
• 5328.

A gRNA or sgRNA comprising;

• a) SEQ ID NO:
• 5321 and any one of SEQ ID NOs:
• 5267-5269;
• b) SEQ ID NO:
• 5323 and any one of SEQ ID NOs:
• 5267-5269;
• c) SEQ ID NO:
• 5325 and any one of SEQ ID NOs:
• 5267-5269;
• d) SEQ ID NO:
• 5327 and any one of SEQ ID NOs:
• 5267-5269;
• or
• e) SEQ ID NO:
• 5328 and any one of SEQ ID NOs:
• 5267-5269.

A method for treating a patient with an USH2A gene containing an IVS40 mutation, the method comprising:

• administering an endonuclease selected from the group consisting of a Cas9 endonuclease and nickases and a Cfp1 endonuclease and a gRNA or sgRNA to eye cells of the patient, wherein the gRNA or sgRNA comprises SEQ ID NO:
• 5321, SEQ ID NO:
• 5323, SEQ ID NO:
• 5325, SEQ ID NO:
• 5327, or SEQ ID NO:
• 5328.

A method for treating a patient with an USH2A gene containing an IVS40 mutation, the method comprising:

• administering an endonuclease selected from the group consisting of a Cas9 endonuclease and nickases and a Cfp1 endonuclease and a gRNA or sgRNA to eye cells of the patient, wherein the gRNA or sgRNA comprises
• a) SEQ ID NO:
• 5321 and any one of SEQ ID NOs:
• 5267-5269;
• b) SEQ ID NO:
• 5323 and any one of SEQ ID NOs:
• 5267-5269;
• c) SEQ ID NO:
• 5325 and any one of SEQ ID NOs:
• 5267-5269;
• d) SEQ ID NO:
• 5327 and any one of SEQ ID NOs:
• 5267-5269;
• or
• e) SEQ ID NO:
• 5328 and any one of SEQ ID NOs:
• 5267-5269.

A method for treating a patient with an USH2A gene containing an IVS40 mutation, the method comprising:

• administering an endonuclease selected from the group consisting of Cas9 and Cfp1 endonucleases and nickases and a first gRNA or sgRNA and second gRNA or sgRNA to eye cells of the patient, wherein
• a) the first gRNA or sgRNA comprises SEQ ID NO:
• 5295 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5279;
• b) the first gRNA or sgRNA comprises SEQ ID NO:
• 5294 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5300;
• c) the first gRNA or sgRNA comprises SEQ ID NO:
• 5295 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5300;
• d) the first gRNA or sgRNA comprises SEQ ID NO:
• 5290 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5300;
• or
• e) the first gRNA or sgRNA comprises SEQ ID NO:
• 5277 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5300.

A method for treating a patient with an USH2A gene containing an IVS40 mutation, the method comprising:

• administering an endonuclease selected from the group consisting of Cas9 and Cfp1 endonucleases and nickases and a first gRNA or sgRNA and second gRNA or sgRNA to eye cells of the patient, wherein
• a) a first gRNA or sgRNA comprises SEQ ID NO:
• 5452 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5449;
• b) a first gRNA or sgRNA comprises SEQ ID NO:
• 5453 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5449;
• c) a first gRNA or sgRNA comprises SEQ ID NO:
• 5455 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5457;
• d) a first gRNA or sgRNA comprises SEQ ID NO:
• 5452 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5451;
• or
• e) a first gRNA or sgRNA comprises SEQ ID NO:
• 5448 and the second gRNA or sgRNA comprises SEQ ID NO:
• 5449.