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publicationDate 2016-03-31-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2016049230-A1
titleOfInvention Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
abstract Adeno-associated virus (AAV) Clade F vectors or AAV vector variants (relative to AAV9) for precise editing of the genome of a cell and methods and kits thereof are provided. Targeted genome editing using the AAV Clade F vectors or AAV vector variants provided herein occurred at frequencies that were shown to be 1,000 to 100,000 fold more efficient than has previously been reported. Also provided are methods of treating a disease or disorder in a subject by editing the genome of a cell of the subject via transducing the cell with an AAV Clade F vector or AAV vector variant as described herein and further transplanting the transduced cell into the subject to treat the disease or disorder of the subject. Also provided herein are methods of treating a disease or disorder in a subject by in vivo genome editing by directly administering the AAV Clade F vector or AAV vector variant as described herein to the subject.
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http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID14071
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http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA0A5F4DEZ7
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http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID81822
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http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID2996
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http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP67973
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http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID3699841
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP67974
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA5PJB2
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP35833
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ9NDE7
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID32051
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID13645
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP67968
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID448365594
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http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID45875
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http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP67970
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID461999
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http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID419590486
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Total number of triples: 444.