ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION AND METHODS OF USE THEREOF
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.
1 . A polynucleotide comprising: a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130; or a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127.
2 . The polynucleotide of claim 1 , comprising a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130.
3 . The polynucleotide of claim 1 , comprising the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130.
4 . The polynucleotide of claim 1 , comprising the nucleotide sequence of SEQ ID NO: 130.
5 . The polynucleotide of claim 1 , comprising a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127.
6 . The polynucleotide of claim 1 , comprising the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127.
7 . The polynucleotide of claim 1 , comprising the nucleotide sequence of SEQ ID NO: 127.
8 . A vector comprising a polynucleotide that comprises:
a) a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;
b) the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;
c) the nucleotide sequence of SEQ ID NO: 130;
d) a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127;
e) the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127; or
f) the nucleotide sequence of SEQ ID NO: 127.
9 . The vector of claim 8 , which is an adeno-associated virus (AAV) vector.
10 . A cell comprising a polynucleotide that comprises:
a) a nucleotide sequence that is at least 99% identical to the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;
b) the nucleotide sequence set forth in positions 7-871 of SEQ ID NO: 130;
c) the nucleotide sequence of SEQ ID NO: 130;
d) a nucleotide sequence that is at least 96% identical to the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127;
e) the nucleotide sequence set forth in positions 7-589 of SEQ ID NO: 127; or
f) the nucleotide sequence of SEQ ID NO: 127.
11 . A cell comprising the vector of claim 8 .
12 . A cell comprising the vector of claim 9 .
13 . The cell of claim 10 , which is a mammalian cell or an insect cell.
14 . The cell of claim 11 , which is a mammalian cell or an insect cell.
15 . The cell of claim 12 , which is a mammalian cell or an insect cell.