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US 12,753,418 B2Granted
Compositions and methods for the treatment of hemophilia
AssigneePoseida Therapeutics, Inc.
InventorsJulian David Down, Xinggang Liu, Jingjing Jiang
Life of this patent
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Abstract
The present disclosure is directed to compositions and methods for the treatment of hemophilia. The compositions and methods can comprise an adeno-associated virus (AAV) piggyBac polynucleotide comprising a transgene. The transgene may comprise ornithine Factor VIII (FVIII) or Factor IX (FIX).
Claims15
An 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:
- 17;
- b) a first piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 19;
- c) a first insulator sequence comprising the nucleic acid sequence of SEQ ID NO:
- 5;
- d) at least one promoter sequence comprising the nucleic acid sequence of SEQ ID NO:
- 12;
- e) a first transgene sequence comprising the nucleic acid sequence of SEQ ID NO:
- 23;
- f) a polyA sequence comprising the nucleic acid sequence of SEQ ID NO:
- 22;
- g) a second insulator sequence comprising the nucleic acid sequence of SEQ ID NO:
- 20;
- h) a second piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 21;
- i) a DNA spacer sequence comprising the nucleic acid sequence SEQ ID NO:
- 28;
- and
- j) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 18.
18 more elements
An 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:
- 1;
- b) a first piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 3;
- c) a first insulator sequence comprising the nucleic acid sequence of SEQ ID NO:
- 5;
- d) at least one promoter sequence comprising the nucleic acid sequence of SEQ ID NO:
- 11;
- e) at least one transgene sequence comprising the nucleic acid sequence of SEQ ID NO:
- 23;
- f) a polyA sequence comprising the nucleic acid sequence of SEQ ID NO:
- 14;
- g) a second insulator sequence comprising the nucleic acid sequence of SEQ ID NO:
- 6;
- h) a second piggyBac ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 4;
- and
- i) a second AAV ITR sequence comprising the nucleic acid sequence of SEQ ID NO:
- 2.
16 more elements
Dependencies are inferred from the claim text.