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AAV

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Sources of off-target expression from recombinase-dependent AAV vectors and mitigation with cross-over insensitive ATG-out vectors
Kyle B. Fischer, Hannah K. Collins, and Edward M. Callaway

PNAS December 26, 2019 116 (52) 27001-27010; first published December 16, 2019. https://doi.org/10.1073/pnas.1915974116
Open Access
A single combination gene therapy treats multiple age-related diseases
Noah Davidsohn, Matthew Pezone, Andyna Vernet, Amanda Graveline, Daniel Oliver, Shimyn Slomovic, Sukanya Punthambaker, Xiaoming Sun, Ronglih Liao, Joseph V. Bonventre, and George M. Church

PNAS November 19, 2019 116 (47) 23505-23511; first published November 4, 2019. https://doi.org/10.1073/pnas.1910073116
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AAV2/9-mediated overexpression of MIF inhibits SOD1 misfolding, delays disease onset, and extends survival in mouse models of ALS
Marcel F. Leyton-Jaimes, Joy Kahn, and Adrian Israelson

PNAS July 16, 2019 116 (29) 14755-14760; first published July 1, 2019. https://doi.org/10.1073/pnas.1904665116
Open Access
AAV cis-regulatory sequences are correlated with ocular toxicity
Wenjun Xiong, David M. Wu, Yunlu Xue, Sean K. Wang, Michelle J. Chung, Xuke Ji, Parimal Rana, Sophia R. Zhao, Shuyi Mai, and Constance L. Cepko

PNAS March 19, 2019 116 (12) 5785-5794; first published March 4, 2019. https://doi.org/10.1073/pnas.1821000116
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Modeling Parkinson’s disease pathology by combination of fibril seeds and α-synuclein overexpression in the rat brain
Poonam Thakur, Ludivine S. Breger, Martin Lundblad, Oi Wan Wan, Bengt Mattsson, Kelvin C. Luk, Virginia M. Y. Lee, John Q. Trojanowski, and Anders Björklund

PNAS September 26, 2017 114 (39) E8284-E8293; first published September 12, 2017. https://doi.org/10.1073/pnas.1710442114
Open Access
Critical role of RAGE and HMGB1 in inflammatory heart disease
Anna Bangert, Martin Andrassy, Anna-Maria Müller, Mariella Bockstahler, Andrea Fischer, Christian H. Volz, Christoph Leib, Stefan Göser, Sevil Korkmaz-Icöz, Stefan Zittrich, Andreas Jungmann, Felix Lasitschka, Gabriele Pfitzer, Oliver J. Müller, Hugo A. Katus, and Ziya Kaya

PNAS January 12, 2016 113 (2) E155-E164; first published December 29, 2015. https://doi.org/10.1073/pnas.1522288113
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Alleviation of off-target effects from vector-encoded shRNAs via codelivered RNA decoys
Stefan Mockenhaupt, Stefanie Grosse, Daniel Rupp, Ralf Bartenschlager, and Dirk Grimm

PNAS July 28, 2015 112 (30) E4007-E4016; first published July 13, 2015. https://doi.org/10.1073/pnas.1510476112
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Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I
Christian Hinderer, Peter Bell, Brittney L. Gurda, Qiang Wang, Jean-Pierre Louboutin, Yanqing Zhu, Jessica Bagel, Patricia O’Donnell, Tracey Sikora, Therese Ruane, Ping Wang, Mark E. Haskins, and James M. Wilson

PNAS October 14, 2014 111 (41) 14894-14899; first published September 29, 2014. https://doi.org/10.1073/pnas.1413645111
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Vectored antibody gene delivery protects against Plasmodium falciparum sporozoite challenge in mice
Cailin Deal, Alejandro B. Balazs, Diego A. Espinosa, Fidel Zavala, David Baltimore, and Gary Ketner

PNAS August 26, 2014 111 (34) 12528-12532; first published August 11, 2014. https://doi.org/10.1073/pnas.1407362111

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