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Molecular Therapy. Methods & Clinical Development|August 4, 2018
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus MacaquesJuliette Hordeaux, Christian Hinderer, Tamara Goode, et al.
Cancer Discovery|December 31, 2015
Hif1a Deletion Reveals Pro-Neoplastic Function of B Cells in Pancreatic NeoplasiaKyoung Eun Lee, Michelle Spata, Lauren J Bayne, et al.
Human Gene Therapy|April 25, 2019
Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus MonkeysJuliette Hordeaux, Christian Hinderer, Elizabeth L Buza, et al.
Journal of Immunology (Baltimore, Md. : 1950)|September 9, 2005
Characterization of latent membrane protein 2 specificity in CTL lines from patients with EBV-positive nasopharyngeal carcinoma and lymphomaKarin C Straathof, Ann M Leen, Elizabeth L Buza, et al.
Hepatology (Baltimore, Md.)|April 22, 2016
Genetic lineage tracing analysis of the cell of origin of hepatotoxin-induced liver tumors in miceSoona Shin, Kirk J Wangensteen, Monica Teta-Bissett, et al.
Human Gene Therapy. Clinical Development|January 30, 2019
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson DiseaseJenny A Greig, Jayme M L Nordin, Melanie K Smith, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 20, 2021
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9Lili Wang, Camilo Breton, Claude C Warzecha, et al.
Science Translational Medicine|November 12, 2020
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primatesJuliette Hordeaux, Elizabeth L Buza, Brianne Jeffrey, et al.
Cellular and Molecular Gastroenterology and Hepatology|January 8, 2016
Spontaneous Pancreatitis Caused by Tissue-Specific Gene Ablation of Hhex in MiceMark J Ferreira, Lindsay B McKenna, Jia Zhang, et al.
Molecular Therapy. Methods & Clinical Development|November 24, 2022
Erratum: Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectorsMakoto Horiuchi, Christian J Hinderer, Jenny A Greig, et al.
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