Abstract：
:Recombinant adeno-associated viruses (rAAVs) are commonly used vehicles for in vivo gene transfer. However, the tropism repertoire of naturally occurring AAVs is limited, prompting a search for novel AAV capsids with desired characteristics. Here we describe a capsid selection method, called Cre recombination-based AAV targeted evolution (CREATE), that enables the development of AAV capsids that more efficiently transduce defined Cre-expressing cell populations in vivo. We use CREATE to generate AAV variants that efficiently and widely transduce the adult mouse central nervous system (CNS) after intravenous injection. One variant, AAV-PHP.B, transfers genes throughout the CNS with an efficiency that is at least 40-fold greater than that of the current standard, AAV9 (refs. 14,15,16,17), and transduces the majority of astrocytes and neurons across multiple CNS regions. In vitro, it transduces human neurons and astrocytes more efficiently than does AAV9, demonstrating the potential of CREATE to produce customized AAV vectors for biomedical applications.
journal_name
Nat Biotechnoljournal_title
Nature biotechnologyauthors
Deverman BE,Pravdo PL,Simpson BP,Kumar SR,Chan KY,Banerjee A,Wu WL,Yang B,Huber N,Pasca SP,Gradinaru Vdoi
10.1038/nbt.3440subject
Has Abstractpub_date
2016-02-01 00:00:00pages
204-9issue
2eissn
1087-0156issn
1546-1696pii
nbt.3440journal_volume
34pub_type
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