Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity
AssigneeGenecraft Inc.
InventorsSuk Chul Bae, You Soub Lee, Xinzi Chi, Tae Geun Park, Woo-Jin Kim
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Abstract
Described herein is an adeno-associated virus (AAV) complex platform including an asymmetrically modified inverted terminal repeat (ITR). The AAV complex has advantages of increased productivity and expression efficiency of a transgene, and decreased genotoxicity, by having an asymmetric ITR in which any one of two ITRs is modified. Also, described herein is a composition comprising the adeno-associated virus complex and a method of gene therapy.
Claims9
An adeno-associated virus (AAV) complex, comprising a polynucleotide sequence encoding a transgene between a first inverted terminal repeat (ITR) and a second ITR,
- wherein the first ITR is not modified and the second ITR is modified,
- wherein in the second ITR, all or part of a stem-loop structure, which is formed of rep-binding element (RBE), RBE′, A, A′, B, B′, C, C′, and D regions, is modified,
- wherein the modified ITR exists as an open-end without forming any of the stem region and the loop region of the stem-loop structure.
Dependencies are inferred from the claim text.