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US 11,027,000 B2Granted
AADC polynucleotides for the treatment of Parkinson's disease
AssigneeVoyager Therapeutics, Inc.
InventorsRobert Kotin, Adrian Philip Kells, Bernard Ravina
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Abstract
The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.
Claims26
A polynucleotide comprising:
- a) a 5′ inverted terminal repeat (ITR), wherein said 5′ ITR is 141 nucleotides in length,
- b) a cytomegalovirus (CMV) sequence region comprising from 5′ to 3′ a CMV enhancer or a fragment thereof comprising at least 250 nucleotides, and a CMV promoter or a fragment thereof comprising at least 150 nucleotides,
- c) an immediate early 1 (IE1) sequence region comprising an IE1 exon1 or a fragment thereof of at least 50 nucleotides, and an IE1 intron 1 or a fragment thereof of at least 20 nucleotides,
- d) a human beta globin (HB) sequence region comprising an HB intron 2 or a fragment thereof of at least 250 nucleotides, and an HB exon 3 or a fragment thereof of at least 40 nucleotides,
- e) a polynucleotide sequence encoding Aromatic L-Amino Acid Decarboxylase (AADC) or an isoform thereof,
- f) a poly(A) signal sequence region comprising a human growth hormone (hGH) poly(A) signal or a fragment thereof of at least 200 nucleotides, and
- g) a 3′ ITR, wherein said 3′ ITR is 141 nucleotides in length;
- wherein the polynucleotide comprises a nucleotide sequence at least 95% identical to SEQ ID NO:
- 17.
6 more elements
Dependencies are inferred from the claim text.