Methods and Compositions for the Adar-Mediated Editing of Argininosuccinate Synthetase (ASS1)
AssigneeKorro Bio, Inc.
InventorsMatthew Blair Jarpe, Mallikarjuna Reddy Putta, Andrew Fraley
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Abstract
The present invention relates to methods and compositions for editing an ASS1 polynucleotide, e.g., an ASS1 polynucleotide comprising a SNP associated with Citrullinemia Type 1. The invention also relates to methods and compositions for treating or preventing Citrullinemia Type 1 in a subject.
Claims31
A method of editing an ASS1 polynucleotide comprising a single nucleotide polymorphism (SNP) associated with Citrullinemia Type 1, the method comprising contacting the ASS1 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 Citrullinemia Type 1, thereby editing the ASS1 polynucleotide.
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A method of treating Citrullinemia Type 1 in a subject in need thereof, the method comprising
- identifying a subject with a single nucleotide polymorphism (SNP) associated with Citrullinemia Type 1 in an ASS1 polynucleotide;
- contacting the ASS1 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 Citrullinemia Type 1, thereby treating the subject.
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Dependencies are inferred from the claim text.