Compositions and methods for using engineered deubiquitinases for probing ubiquitin-dependent cellular processes
AssigneeThe Trustees of Columbia University in the City of New York
InventorsHenry M. Colecraft, Scott Kanner
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Abstract
The present disclosure provides, inter alia, a recombinant engineered deubiquitinase (DUB) and methods for treating or ameliorating an inherited ion channelopathy, such as long QT syndrome, Brugada syndrome, or cystic fibrosis, in a subject. Further provided are methods for screening mutations causing such inherited ion channelopathies for a trafficking-deficient mutation that is treatable by the recombinant engineered DUB disclosed herein.
Claims30
A method of treating or ameliorating the effects of a disease in a human subject, the method comprising administering to the subject a nucleic acid molecule encoding a recombinant engineered deubiquitinase (DUB) comprising:
- a) a catalytic unit comprising the catalytic domain of a deubiquitinase;
- b) a protein binder comprising an antibody, or antigen binding fragment thereof, that specifically binds a target substrate protein for deubiquitination by the engineered DUB;
- and
- c) a variable linker between the catalytic unit and the protein binder, and
- wherein the disease is cystic fibrosis, long QT syndrome, Brugada syndrome, Bartter Syndrome, or epilepsy.
Dependencies are inferred from the claim text.