Compositions and methods for modulating gene expression
AssigneeKlogenix LLC
InventorsCi-Di Chen, Ella Zeldich, Carmela Abraham
Life of this patent
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Abstract
The present disclosure relates to compositions and methods for modulating gene expression and in particular to compositions and methods for increasing expression of Klotho. For example, the present disclosure provides methods for increasing expression of a Klotho gene in a human cell comprising introducing into the cell a CRISPR enzyme and a guide RNA comprising a guide sequence that is substantially complementary to a target sequence within or near the Klotho gene, wherein the guide RNA or the CRISPR enzyme associates with a transcriptional activation domain.
Claims33
A method of increasing expression of a Klotho gene in a human cell, the method comprising introducing into the cell:
- a CRISPR enzyme;
- and
- a guide RNA comprising a guide sequence that is at least 90% identical to SEQ ID NO. 1 or SEQ ID NO. 3, wherein the guide sequence is substantially complementary to a target sequence located within a region between the Klotho gene translation start site and 4000 nucleotides upstream of the Klotho gene translation start site, wherein the guide RNA associates with a transcriptional activation domain in the cell to thereby increase expression of the Klotho gene.
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A method of increasing expression of a Klotho gene in a human cell, the method comprising introducing into the cell:
- a guide RNA comprising a guide sequence that is at least 90% identical to SEQ ID NO. 1 or SEQ ID NO. 3, wherein the guide sequence is substantially complementary to a target sequence located within a region between the Klotho gene translation start site and 4000 nucleotides upstream of the Klotho gene translation start site;
- and
- a CRISPR enzyme, wherein the CRISPR enzyme comprises or is attached to a transcriptional activation domain.
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A method of treating a kidney disorder in a human subject the method comprising administering to the subject:
- a CRISPR enzyme;
- and
- a guide RNA comprising a guide sequence that is at least 90% identical to SEQ ID NO. 1 or SEQ ID NO. 3, wherein the guide sequence is substantially complementary to a target sequence located within a region between a Klotho gene translation start site and 4000 nucleotides upstream of the Klotho gene translation start site in the subject, wherein the guide RNA associates with a transcriptional activation domain in a cell of the subject and thereby increases expression of the Klotho gene.
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Dependencies are inferred from the claim text.