@article{c67247c2b11b4558b18cd163ee08a605,
title = "Protease-Activatable Adeno-Associated Virus Vector for Gene Delivery to Damaged Heart Tissue",
abstract = "Guenther et al. have developed a protease-activatable AAV provector to target diseased tissue microenvironments exhibiting elevated extracellular protease activity. The provector delivers transgenes to high-MMP-activity regions of the damaged heart following a myocardial infarction, with concomitant decreased delivery to many off-target organs, including the liver.",
keywords = "AAV, activatable, adeno-associated virus, cardiac gene therapy, gene delivery, gene therapy, inflammation targeting, matrix metalloproteinase, myocardial infarction, provector, stimulus responsive, viral vector",
author = "Guenther, {Caitlin M.} and Brun, {Mitchell J.} and Bennett, {Antonette D.} and Ho, {Michelle L.} and Weitong Chen and Banghe Zhu and Michael Lam and Momona Yamagami and Sunkuk Kwon and Nilakshee Bhattacharya and Duncan Sousa and Evans, {Annicka C.} and Julie Voss and Sevick-Muraca, {Eva M.} and Mavis Agbandje-McKenna and Junghae Suh",
note = "Funding Information: This material is based on work supported by the NIH (grant numbers R21HL126053, R01HL138126, and R01CA207497), the American Heart Association (grant numbers 13GRNT14420044 and 15GRNT23070007), and a Dunn Foundation grant to J.S.; an American Heart Association Predoctoral Fellowship (16PRE30690006) to C.M.G.; the NIH (R01GM082946) to M.A.-M.; and National Science Foundation Fellowships to A.C.E. (2015197891) and W.C. (2018253392). We acknowledge the University of North Carolina at Chapel Hill Gene Therapy Center Vector Core for providing us with pXX6-80 and scAAV2-CMV-GFP and the University of Pennsylvania Vector Core for providing us with pAAV2/9. We thank the Electron microscopy core of the University of Florida (UF) Interdisciplinary Center for Biotechnology Research (ICBR) for access to electron microscopes utilized for negative stain electron microscopy and cryoelectron microscopy (cryo-EM) data collection. The Spirit and TF20 cryoelectron microscopes were provided by the UF College of Medicine (COM) and Division of Sponsored Programs (DSP). Data collection at Florida State University was made possible by NIH grants S10 OD018142-01 (purchase of a direct electron camera for the Titan-Krios at FSU, P.I. Taylor), S10 RR025080-01 (purchase of an FEI Titan Krios for 3-D EM, P.I. Taylor), and U24 GM116788 (The Southeastern Consortium for Microscopy of MacroMolecular Machines, P.I. Taylor). Funding Information: This material is based on work supported by the NIH (grant numbers R21HL126053 , R01HL138126 , and R01CA207497 ), the American Heart Association (grant numbers 13GRNT14420044 and 15GRNT23070007 ), and a Dunn Foundation grant to J.S.; an American Heart Association Predoctoral Fellowship ( 16PRE30690006 ) to C.M.G.; the NIH ( R01GM082946 ) to M.A.-M.; and National Science Foundation Fellowships to A.C.E. ( 2015197891 ) and W.C. ( 2018253392 ). We acknowledge the University of North Carolina at Chapel Hill Gene Therapy Center Vector Core for providing us with pXX6-80 and scAAV2-CMV-GFP and the University of Pennsylvania Vector Core for providing us with pAAV2/9. We thank the Electron microscopy core of the University of Florida (UF) Interdisciplinary Center for Biotechnology Research (ICBR) for access to electron microscopes utilized for negative stain electron microscopy and cryoelectron microscopy (cryo-EM) data collection. The Spirit and TF20 cryoelectron microscopes were provided by the UF College of Medicine (COM) and Division of Sponsored Programs (DSP). Data collection at Florida State University was made possible by NIH grants S10 OD018142-01 (purchase of a direct electron camera for the Titan-Krios at FSU, P.I. Taylor), S10 RR025080-01 (purchase of an FEI Titan Krios for 3-D EM, P.I. Taylor), and U24 GM116788 (The Southeastern Consortium for Microscopy of MacroMolecular Machines, P.I. Taylor). Publisher Copyright: {\textcopyright} 2019 The American Society of Gene and Cell Therapy",
year = "2019",
month = mar,
day = "6",
doi = "10.1016/j.ymthe.2019.01.015",
language = "English (US)",
volume = "27",
pages = "611--622",
journal = "Molecular Therapy",
issn = "1525-0016",
publisher = "Nature Publishing Group",
number = "3",
}