Genomic insulator elements and uses thereof
AssigneeUniversity of Washington
InventorsGeorge Stamatoyannopoulos, John Stamatoyannopoulos
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Abstract
Provided herein are methods for identifying high potency genomic insulator elements that can be used in a vector composition e.g., that are useful for preventing unwanted expression of neighboring genes, such as proto-oncogenes, when administered to a subject in need thereof. Also provided herein are methods for treating disease and methods for administering a nucleic acid to a subject using such vectors.
Claims26
A viral vector comprising:
- a cassette comprising a nucleic acid sequence encoding a therapeutic agent driven by a promoter regulated by an enhancer,
- wherein the cassette is bracketed by genomic insulator elements,
- wherein each of the genomic insulator elements comprises a CTCF binding site core sequence selected from the group consisting of:
- (SEQ ID NO.:
- 2)
- CCACCAGGGGGAGC,
- (SEQ ID NO.:
- 3)
- TCAGTAGAGGGCGC,
- (SEQ ID NO.:
- 4)
- CCACTAGGGGGCAG,
- (SEQ ID NO.:
- 5)
- CAGCAGAGGGCGCT,
- and
- (SEQ ID NO.:
- 6)
- CAGTAGAGGGCGCT.
A viral vector comprising a cassette comprising a nucleic acid sequence encoding a therapeutic agent driven by a promoter regulated by an enhancer, wherein the cassette is bracketed by genomic insulator elements, and wherein each of the genomic insulator elements comprises a CTCF binding site core sequence of SEQ
- (SEQ ID NO.:
- 1)
- CACCAGGTGGCGCT.
Dependencies are inferred from the claim text.