CPC C12N 15/86 (2013.01) [A61K 9/0078 (2013.01); A61K 9/12 (2013.01); A61P 11/00 (2018.01); C07K 14/005 (2013.01); C07K 14/4712 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14141 (2013.01); C12N 2750/14142 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14151 (2013.01)] | 28 Claims |
1. A method of delivering a heterologous nucleic acid to a lung cell in a primate comprising administering to the primate a composition comprising a recombinant adeno-associated virus (rAAV) vector, said rAAV vector comprising (i) a capsid comprising a capsid protein comprising the amino acid sequence set forth as SEQ ID NO:12 or an amino acid sequence at least 90% identical to SEQ ID NO:12 and comprising a Thr at amino acid 469 and an Ala at amino acid 598 based on the amino acid numbering set forth in SEQ ID NO: 12 and (ii) a heterologous nucleic acid comprising a nucleotide sequence encoding a gene product, wherein the step of administering comprises pulmonary, endobronchial, intranasal, intratracheal, and/or intrabronchial administration.
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16. A nucleic acid sequence comprising a nucleotide sequence set forth in SEQ ID NO:43 or a sequence at least 80% identical thereto.
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18. A pharmaceutical composition comprising an rAAV vector, said rAAV vector comprising (i) a capsid comprising a capsid protein of SEQ ID NO:12 or an amino acid sequence at least 90% identical to SEQ ID NO:12 and comprising a Thr at amino acid 469 and an Ala at amino acid 598 based on the amino acid numbering set forth in SEQ ID NO: 12 and (ii) a heterologous nucleic acid comprising a nucleotide sequence encoding a CFTR or a biologically active truncated CFTR protein lacking amino acids 708-759 of the human CFTR protein sequence, said nucleotide sequence operably linked to an expression control sequence.
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23. A pharmaceutical composition comprising an rAAV virus, wherein the rAAV virus is formulated in a buffer comprising about 10 mM to about 50 mM citrate, about 70 mM to about 150 mM NaCl and a surfactant and having a pH of about 5.0 to about 7.0.
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27. A method of treating cystic fibrosis in a primate comprising administering to the primate a pharmaceutical composition according to claim 18, wherein the step of administering comprises pulmonary, endobronchial, intranasal, intratracheal, and/or intrabronchial administration.
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