Method of regulating cftr expression and processing
AssigneeUniversity of Iowa Research Foundation
InventorsPaul B. McCray, Shyam Ramachandran
Life of this patent
Estimating the term…
Abstract
The present invention relates to methods of reducing ΔF508-CFTR ubiquitination or degradation, or increasing ΔF508-CFTR processing or function in a CF cell comprising contacting the cell with a therapeutic agent that inhibits NEDD8, FBXO2, and/or SYVN1 expression in the cell.
Claims21
A method of reducing ΔF508-CFTR ubiquitination or degradation, or increasing ΔF508-CFTR processing or function in a cystic fibrosis (CF) cell comprising contacting the cell with a therapeutic agent, wherein the therapeutic agent comprises one or more of
- (a) a NEDD8 therapeutic agent that inhibits NEDD8 expression in the cell,
- (b) a FBXO2 therapeutic agent that inhibits FBXO2 expression in the cell,
- (c) a SYVN1 therapeutic agent that inhibits SYVN1 expression and a AHSA1 therapeutic agent that inhibits AHSA1 expression in the cell, or
- (d) a therapeutic agent that inhibits SYVN1 expression in the cell.
A method of treating a subject having CF comprising administering to the subject an effective amount of a therapeutic agent to alleviate the symptoms of CF, wherein the agent comprises
- (a) an anti-NEDD8 RNAi molecule, and/or an anti-NEDD8 antisense oligonucleotide (ASO) or other agent that suppresses NEDD8 expression, or a small molecule drug that interferes with NEDD8 activity or whose actions mimic the biological effects of NEDD8 suppression, and/or
- (b) an anti-FBXO2 RNAi molecule, and/or an anti-FBXO2 antisense oligonucleotide (ASO) or other agent that suppresses FBXO2 expression, or a small molecule drug that interferes with FBXO2 activity or whose actions mimic the biological effects of FBXO2 suppression;
- and/or
- (c) an anti-SYVN1 RNAi molecule, and/or an anti-SYVN1 antisense oligonucleotide (ASO) or other agent that suppresses SYVN1 expression, or a small molecule drug that interferes with SYVN1 activity or whose actions mimic the biological effects of SYVN1 suppression.
A pharmaceutical composition for treatment of cystic fibrosis, comprising
- (a) an anti-NEDD8 RNAi molecule, and/or an anti-NEDD8 antisense oligonucleotide (ASO) or other agent that suppresses NEDD8 expression, or a small molecule drug that interferes with NEDD8 activity or whose actions mimic the biological effects of NEDD8 suppression, and/or
- (b) an anti-FBXO2 RNAi molecule, and/or an anti-FBXO2 antisense oligonucleotide (ASO) or other agent that suppresses FBXO2 expression, or a small molecule drug that interferes with FBXO2 activity or whose actions mimic the biological effects of FBXO2 suppression;
- and/or
- (c) an anti-SYVN1 RNAi molecule, and/or an anti-SYVN1 antisense oligonucleotide (ASO) or other agent that suppresses SYVN1 expression, or a small molecule drug that interferes with SYVN1 activity or whose actions mimic the biological effects of SYVN1 suppression, for use in treating CF.
Dependencies are inferred from the claim text.