Methods and compositions for the adar-mediated editing of retinochisin 1 (rs1)
AssigneeKorro Bio, Inc.
InventorsMatthew Jarpe, Mallikarjuna Reddy Putta, Andrew Fraley
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Abstract
The present invention relates to methods and compositions for editing an RS1 polynucleotide, e.g., an RS1 polynucleotide comprising a SNP associated with X-linked retinoschisis (XLRS). The invention also relates to methods and compositions for treating or preventing XLRS in a subject.
Claims28
A method of editing an RS1 polynucleotide comprising a single nucleotide polymorphism (SNP) associated with X-linked retinoschisis (XLRS), the method comprising contacting the RS1 polynucleotide with a guide oligonucleotide capable of effecting an adenosine deaminase acting on RNA (ADAR)-mediated adenosine to inosine alteration of the SNP associated with XLRS, thereby editing the RS1 polynucleotide.
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A method of treating XLRS in a subject in need thereof, the method comprising
- contacting the RS1 polynucleotide in a cell of the subject with a guide oligonucleotide capable of effecting an adenosine deaminase acting on RNA (ADAR)-mediated adenosine to inosine alteration of the SNP associated with XLRS, thereby treating the subject.
A method of treating XLRS in a subject in need thereof, the method comprising
- contacting the RS1 polynucleotide in a cell with a guide oligonucleotide capable of effecting an adenosine deaminase acting on RNA (ADAR)-mediated adenosine to inosine alteration of the SNP associated with XLRS, and
- administering the cell to the subject, thereby treating the subject.
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Dependencies are inferred from the claim text.