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US 12,540,335 B2Granted
Compositions and methods for the treatment of metabolic liver disorders
AssigneePoseida Therapeutics, Inc.
InventorsJingjing Jiang, Julian Down, Xianghong Li, Eric M. Ostertag
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Abstract
The present disclosure is directed to compositions and methods for the treatment of Metabolic Liver Disorders. The compositions and methods can comprise an adeno-associated virus (AAV) piggyBac polynucleotide comprising a transgene. The transgene may comprise ornithine transcarbamylase (OTC) or methylmalonyl-CoA mutase (MUT1).
Claims18
An adeno-associated virus (AAV) piggyBac transposon polynucleotide comprising in the 5′ to 3′ direction:
- a) a first AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 93;
- b) a first piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 125;
- c) a first insulator sequence comprising the nucleic acid sequence of SEQ ID NO:
- 7;
- d) at least one promoter sequence comprising the nucleic acid sequence of SEQ ID NO:
- 132;
- e) at least one transgene sequence comprising the nucleic acid sequence of SEQ ID NO:
- 22;
- f) a polyA sequence comprising the nucleic acid sequence of SEQ ID NO:
- 97;
- g) a second insulator sequence comprising the nucleic acid sequence of SEQ ID NO:
- 8;
- h) a second piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 96;
- i) at least one DNA spacer sequence comprising the nucleic acid sequence of SEQ ID NO:
- 130;
- and
- j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 94.
18 more elements
Dependencies are inferred from the claim text.