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AssigneeThe Regents of the University of California
InventorsKathrin Plath, Amy Pandya-Jones, Shawn Tan, Douglas Black, Giolanta Markaki
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Aspects of the disclosure relate to a method for re-activating the X-chromosome in cells comprising administering a SPEN inhibitor to the cell. Further aspects relate to a method for re-activating the X-chromosome in a subject in need thereof, the method comprising administering a SPEN inhibitor to the subject. Yet further aspects relate to a method for treating Rett Syndrome in a subject in need thereof, the method comprising administering a SPEN inhibitor to the subject. Aspects of the disclosure relate to a method for re-activating the X-chromosome in cells comprising administering a PTBP1 inhibitor and a DNA demethylating agent to the cell. Further aspects relate to a method for re-activating the X-chromosome in a subject in need thereof, the method comprising administering a PTBP1 inhibitor and a DNA demethylating agent to the subject. Yet further aspects relate to a method for treating Rett syndrome in a subject in need thereof, the method comprising administering a PTBP1 inhibitor and a DNA demethylating agent to the subject.
A method for re-activating an inactive X-chromosome in a cell, wherein the inactive X-chromosome is associated with Xist-mediated gene silencing, the method comprising contacting the cell with a polypyrimidine tract-binding protein 1 (PTBP1) inhibitor and a DNA demethylating agent, wherein the PTBP1 inhibitor is a nucleic acid inhibitor that is complimentary to an mRNA encoded by PTBP1, and wherein the nucleic acid inhibitor is a small interfering RNA (siRNA), a double stranded RNA (dsRNA), a short hairpin RNA (shRNA), or an antisense oligonucleotide.
A method for treating Rett syndrome or an X-linked disorder in a subject, wherein the subject has an inactive X-chromosome which is associated with Xist-mediated gene silencing, the method comprising administering a polypyrimidine tract-binding protein 1 (PTBP1) inhibitor and a DNA demethylating agent to the subject, wherein the PTBP1 inhibitor is a nucleic acid inhibitor that is complimentary to an mRNA encoded by PTBP1, and wherein the nucleic acid inhibitor is a small interfering RNA (siRNA), a double stranded RNA (dsRNA), a short hairpin RNA (shRNA), or an antisense oligonucleotide.
Dependencies are inferred from the claim text.