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US 2016/0102320 A1Published application
Adenoviral vector encoding human atonal homolog-1 (hath1)
AssigneeGenVec, Inc.
InventorsDouglas E. Brough, Damodar R. Ettyreddy
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Abstract
The invention is directed to a replication-deficient adenoviral vector comprising a nucleic acid sequence encoding a human atonal homolog-1 (Hath1) protein operably linked to a human glial fibrillary acidic protein (GFAP) promoter. The invention also is directed to a composition and method utilizing the adenoviral vector to generate sensory cells in the inner ear of a human.
Claims15
An adenoviral vector comprising:
- (a) a serotype 5 adenoviral genome, except that two base pairs are deleted in the VA RNA I region, one or more endogenous nucleotides of the E1 region are deleted to render an endogenous gene in the E1 region functionally deficient, one or more endogenous nucleotides of the E3 region are deleted, and one or more endogenous nucleotides of the E4 region are deleted to render an endogenous gene in the E4 region functionally deficient, wherein a right side inverted terminal repeat (ITR), an E4 region polyadenylation sequence, and an E4 region promoter are retained,
- (b) an SV40 early polyadenylation sequence,
- (c) a nucleic acid sequence encoding a human atonal homolog-1 (Hath1) protein comprising SEQ ID NO:
- 1,
- (d) a human glial fibrillary acidic protein (GFAP) promoter, and
- (e) a transcriptionally inert spacer (TIS),
- wherein elements (b), (c), and (d) are located in sequence from 3′ to 5′ relative to the adenoviral genome in the E1 region of the adenoviral genome, and wherein element (e) is located in the E4 region of the adenoviral genome between the E4 polyadenylation sequence and the E4 promoter.
4 more elements
Dependencies are inferred from the claim text.