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1. (WO2017070476) SYNTHETIC COMBINATORIAL AAV3 CAPSID LIBRARY

Pub. No.:    WO/2017/070476    International Application No.:    PCT/US2016/058130
Publication Date: Fri Apr 28 01:59:59 CEST 2017 International Filing Date: Sat Oct 22 01:59:59 CEST 2016
IPC: A61K 31/7088
A61K 35/76
A61K 48/00
C07K 14/005
C12N 7/00
C12N 15/63
C12N 15/86
C12N 15/861
C12N 15/864
Applicants: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
Inventors: ZOLOTUKHIN, Sergei
MARSIC, Damien
Title: SYNTHETIC COMBINATORIAL AAV3 CAPSID LIBRARY
Abstract:
Compositions and methods for producing modified AAV Cap genes and combinatorial libraries of chimeric AAV vectors and virions in an AAV serotype 3 background. Selecting for modified AAV3 virions displaying cell- or tissue-specific tropisms differing from WT AAV3. Using the synthetic combinatorial AAV3 capsid libraries for introducing into a selected target host cells one or more nucleic acid molecules useful in diagnostic and/or therapeutic gene-therapy regimens.