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Nature|September 24, 2004
Exogenous control of mammalian gene expression through modulation of RNA self-cleavageLaising Yen, Jennifer Svendsen, Jeng-Shin Lee, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 20, 2011
Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosisHuimin Hu, Elida Gomero, Erik Bonten, et al.
Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|August 24, 2005
Adeno-associated virus vector-mediated systemic delivery of IFN-beta combined with low-dose cyclophosphamide affects tumor regression in murine neuroblastoma modelsChristian J Streck, Paxton V Dickson, Catherine Y C Ng, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 1, 2005
Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate modelsAndrew M Davidoff, John T Gray, Catherine Y C Ng, et al.
Blood|September 18, 2013
Nfix is a novel regulator of murine hematopoietic stem and progenitor cell survivalPer Holmfeldt, Jennifer Pardieck, Anjelica C Saulsberry, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 16, 2008
Globin lentiviral vector insertions can perturb the expression of endogenous genes in beta-thalassemic hematopoietic cellsPhillip W Hargrove, Steven Kepes, Hideki Hanawa, et al.
Human Molecular Genetics|June 23, 2018
TNNT1 nemaline myopathy: natural history and therapeutic frontierMichael D Fox, Vincent J Carson, Han-Zhong Feng, et al.
Molecular Therapy. Methods & Clinical Development|August 17, 2018
Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar SyndromeJenny A Greig, Jayme M L Nordin, Christine Draper, et al.
Blood|November 9, 2006
Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primatesAmit C Nathwani, John T Gray, Jenny McIntosh, et al.
Proceedings of the National Academy of Sciences of the United States of America|July 18, 2008
Soluble receptor-mediated selective inhibition of VEGFR and PDGFRbeta signaling during physiologic and tumor angiogenesisFrank Kuhnert, Betty Y Y Tam, Barbara Sennino, et al.
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