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US 12,203,094 B2Granted
Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
AssigneeHomology Medicines, Inc.
InventorsAlbert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, James Anthony McSwiggen, Jaime Michelle Prout, Danielle Lauren Sookiasian
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Abstract
Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
Claims20
A replication-defective adeno-associated virus (AAV) comprising:
- (a) an AAV capsid;
- and
- (b) a correction genome comprising:
- (i) an editing element for editing a target locus in a human phenylalanine hydroxylase (PAH) gene, wherein the editing element comprises a nucleotide sequence that is at least 90% identical to the nucleotide sequence set forth in SEQ ID NO:
- 35, 83, or 84;
- (ii) a 5′ homology arm located 5′ of the editing element, the 5′ homology arm comprising a nucleotide sequence of at least 100 nucleotides in length, that is at least 90% identical to a nucleic acid sequence contained within the nucleic acid sequence set forth in SEQ ID NO:
- 36, 37, 38, 39, 40, 41, 42, 43, 44, or 115;
- and
- (iii) a 3′ homology arm located 3′ of the editing element, the 3′ homology arm comprising a nucleotide sequence of at least 100 nucleotides in length, that is at least 90% identical to a nucleic acid sequence contained within the nucleic acid sequence set forth in SEQ ID NO:
- 45 or 117.
7 more elements
A polynucleotide comprising the nucleic acid sequence of any one of SEQ ID NO:
- 25, 46-63, 80-82, 116, 118, 119, 131, 132, 135-139, 143, 145, or 146, optionally wherein the polynucleotide is comprised within a vector or host cell.
Dependencies are inferred from the claim text.