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US 2026/0308623 A1Published application
Single plasmid system for aav production
AssigneeSolid Biosciences Inc.
InventorSarath Chandra Mandava
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Abstract
The invention described herein provides an improved system and method for rAAV production. The system of the invention includes a single recombinant DNA vector that comprises a gene of interest (GO I) to be delivered to a target cell, the Rep and Cap coding sequences, as well as the Helper gene coding sequences for rAAV packaging.
Claims27
A recombinant DNA vector comprising:
- (a) a gene-of-interest (GOI) flanked by a 5′ adeno-associated virus (AAV) ITR (such as AAV2 5′ ITR) sequence and a 3′ ITR sequence (such as AAV2 3′ ITR);
- (b) a coding sequence for an AAV Rep (such as AAV2 Rep, or Rep2) compatible with said 5′ AAV ITR and/or said 3′ AAV ITR;
- (c) a coding sequence for an AAV Cap;
- and
- (d) a coding sequence for AAV Helper genes sufficient for enabling AAV packaging;
- wherein the coding sequence for the AAV Rep and the coding sequence for the AAV Cap are within a RepCap cassette comprising an operably-linked RepCap promoter (such as the AAV P5 promoter);
- and,
- wherein the GOI flanked by the 5′ and 3′ ITRs is positioned upstream of (e.g., adjacent to, or immediately 5′ to) the 5′ end of the promoter of the RepCap cassette.
5 more elements
Dependencies are inferred from the claim text.