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US 12,606,846 B2Granted
Materials and methods for the treatment of disorders associated with the IRF2BPL gene
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsNicolas Sebastien Wein, Kathrin Christine Meyer
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Abstract
The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of an Interferon regulatory factor 2 binding protein like (IRF2BPL) disorder.
Claims7
A polynucleotide comprising
- (a) a regulatory control element, wherein the regulatory control element is (i) a pIRF promoter comprising the sequence set forth by SEQ ID NO:
- 9 or SEQ ID NO:
- 10, or (ii) a pIRF promoter comprising the sequence set forth by SEQ ID NO:
- 8 operably linked to a p546 promoter comprising the sequence set forth by SEQ ID NO:
- 5;
- and
- (b) an Interferon regulatory factor 2 binding protein like (IRF2BPL) cDNA sequence operably linked to the regulatory control element.
4 more elements
Dependencies are inferred from the claim text.