Gene therapy clinical trials worldwide to 2017: An update von Ginn, Samantha L., Amaya, Anais K., Alexander, Ian E., Edelstein, Michael, Abedi, Mohammad R.

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Gene therapy clinical trials worldwide to 2012 - an update von Ginn, Samantha L., Alexander, Ian E., Edelstein, Michael L., Abedi, Mohammad R., Wixon, Jo

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model von Lisowski, Leszek, Dane, Allison P., Chu, Kirk, Zhang, Yue, Cunningham, Sharon C., Wilson, Elizabeth M., Nygaard, Sean, Grompe, Markus, Alexander, Ian E., Kay, Mark A.

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The implementation of newborn screening for spinal muscular atrophy: the Australian experience von Kariyawasam, Didu, Russell, Jacqueline S., Wiley, Veronica, Alexander, Ian E., Farrar, Michelle A.

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Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects von Baruteau, Julien, Waddington, Simon N., Alexander, Ian E., Gissen, Paul

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The Transcriptional and Functional Properties of Mouse Epiblast Stem Cells Resemble the Anterior Primitive Streak von Kojima, Yoji, Kaufman-Francis, Keren, Studdert, Joshua B., Steiner, Kirsten A., Power, Melinda D., Loebel, David A.F., Jones, Vanessa, Hor, Angelyn, de Alencastro, Gustavo, Logan, Grant J., Teber, Erdahl T., Tam, Oliver H., Stutz, Michael D., Alexander, Ian E., Pickett, Hilda A., Tam, Patrick P.L.

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Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV‐piggyBac Gene Therapy von Siew, Susan M., Cunningham, Sharon C., Zhu, Erhua, Tay, Szun S., Venuti, Elena, Bolitho, Christine, Alexander, Ian E.

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Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome von Logan, Grant J, Dane, Allison P, Hallwirth, Claus V, Smyth, Christine M, Wilkie, Emilie E, Amaya, Anais K, Zhu, Erhua, Khandekar, Neeta, Ginn, Samantha L, Liao, Sophia H Y, Cunningham, Sharon C, Sasaki, Natsuki, Cabanes-Creus, Martí, Tam, Patrick P L, Russell, David W, Lisowski, Leszek, Alexander, Ian E

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Lymphoid Regeneration from Gene-Corrected SCID-X1 Subject-Derived iPSCs von Menon, Tushar, Firth, Amy L., Scripture-Adams, Deirdre D., Galic, Zoran, Qualls, Susan J., Gilmore, William B., Ke, Eugene, Singer, Oded, Anderson, Leif S., Bornzin, Alexander R., Alexander, Ian E., Zack, Jerome A., Verma, Inder M.

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Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation von Cabanes-Creus, Marti, Liao, Sophia H. Y., Gale Navarro, Renina, Knight, Maddison, Nazareth, Deborah, Lau, Ngee-Soon, Ly, Mark, Zhu, Erhua, Roca-Pinilla, Ramon, Bugallo Delgado, Ricardo, Vicente, Ana F., Baltazar, Grober, Westhaus, Adrian, Merjane, Jessica, Crawford, Michael, McCaughan, Geoffrey W., Unzu, Carmen, González-Aseguinolaza, Gloria, Alexander, Ian E., Pulitano, Carlo, Lisowski, Leszek

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Antigen expression level threshold tunes the fate of CD8 T cells during primary hepatic immune responses von Tay, Szun Szun, Wong, Yik Chun, McDonald, David M, Wood, Nicole A W, Roediger, Ben, Sierro, Frederic, Mcguffog, Claire, Alexander, Ian E, Bishop, G Alex, Gamble, Jennifer R, Weninger, Wolfgang, McCaughan, Geoffrey W, Bertolino, Patrick, Bowen, David G

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Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy von D'Silva, Arlene M., Holland, Sandra, Kariyawasam, Didu, Herbert, Karen, Barclay, Peter, Cairns, Anita, MacLennan, Suzanna C., Ryan, Monique M., Sampaio, Hugo, Smith, Nicholas, Woodcock, Ian R., Yiu, Eppie M., Alexander, Ian E., Farrar, Michelle A.

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Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease von Ng, Roy Chun-Laam, Jian, Min, Ma, Oscar Ka-Fai, Xiang, Ariya Weiman, Bunting, Myriam, Kwan, Jason Shing-Cheong, Wong, Curtis Wai-Kin, Yick, Leung-Wah, Chung, Sookja Kim, Lam, Karen Siu-Ling, Alexander, Ian E, Xu, Aimin, Chan, Koon-Ho

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ACE2 Therapy Using Adeno-associated Viral Vector Inhibits Liver Fibrosis in Mice von Mak, Kai Y, Chin, Ruth, Cunningham, Sharon C, Habib, Miriam R, Torresi, Joseph, Sharland, Alexandra F, Alexander, Ian E, Angus, Peter W, Herath, Chandana B

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Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors von Cunningham, Sharon C, Dane, Allison P, Spinoulas, Afroditi, Alexander, Ian E

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Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys von Baruteau, Julien, Cunningham, Sharon C., Yilmaz, Berna Seker, Perocheau, Dany P., Eaglestone, Simon, Burke, Derek, Thrasher, Adrian J., Waddington, Simon N., Lisowski, Leszek, Alexander, Ian E., Gissen, Paul

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Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method von Rao, Renuka, Farraha, Melad, Logan, Grant J, Igoor, Sindhu, Kok, Cindy Y, Chong, James J. H, Alexander, Ian E, Kizana, Eddy

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Gene therapy for urea cycle defects: An update from historical perspectives to future prospects von Duff, Claire, Alexander, Ian E., Baruteau, Julien

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AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia von Graves, Lara E., van Dijk, Eva B., Zhu, Erhua, Koyyalamudi, Sundar, Wotton, Tiffany, Sung, Dinah, Srinivasan, Shubha, Ginn, Samantha L., Alexander, Ian E.

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Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno‐associated virus/piggyBac transposase gene delivery system von Cunningham, Sharon C., Siew, Susan M., Hallwirth, Claus V., Bolitho, Christine, Sasaki, Natsuki, Garg, Gagan, Michael, Iacovos P., Hetherington, Nicola A., Carpenter, Kevin, de Alencastro, Gustavo, Nagy, Andras, Alexander, Ian E.

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