Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy von Wilson, James M, Flotte, Terence R

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The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice von Hordeaux, Juliette, Wang, Qiang, Katz, Nathan, Buza, Elizabeth L., Bell, Peter, Wilson, James M.

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Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN von Hinderer, Christian, Katz, Nathan, Buza, Elizabeth L, Dyer, Cecilia, Goode, Tamara, Bell, Peter, Richman, Laura K, Wilson, James M

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Scalable mRNA and siRNA Lipid Nanoparticle Production Using a Parallelized Microfluidic Device von Shepherd, Sarah J, Warzecha, Claude C, Yadavali, Sagar, El-Mayta, Rakan, Alameh, Mohamad-Gabriel, Wang, Lili, Weissman, Drew, Wilson, James M, Issadore, David, Mitchell, Michael J

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Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses von Calcedo, Roberto, Vandenberghe, Luk H., Gao, Guangping, Lin, Jianping, Wilson, James M.

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Humoral Immune Response to AAV von Calcedo, Roberto, Wilson, James M

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Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency von Wilson, James M.

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Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology von Hordeaux, Juliette, Buza, Elizabeth L, Dyer, Cecilia, Goode, Tamara, Mitchell, Thomas W, Richman, Laura, Denton, Nathan, Hinderer, Christian, Katz, Nathan, Schmid, Ralf, Miller, Rod, Choudhury, Gourav R, Horiuchi, Makoto, Nambiar, Kalyani, Yan, Hanying, Li, Mingyao, Wilson, James M

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History Lesson for Stem Cells von Wilson, James M

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Universal protection against influenza infection by a multidomain antibody to influenza hemagglutinin von Laursen, Nick S, Friesen, Robert H E, Zhu, Xueyong, Jongeneelen, Mandy, Blokland, Sven, Vermond, Jan, van Eijgen, Alida, Tang, Chan, van Diepen, Harry, Obmolova, Galina, van der Neut Kolfschoten, Marijn, Zuijdgeest, David, Straetemans, Roel, Hoffman, Ryan M B, Nieusma, Travis, Pallesen, Jesper, Turner, Hannah L, Bernard, Steffen M, Ward, Andrew B, Luo, Jinquan, Poon, Leo L M, Tretiakova, Anna P, Wilson, James M, Limberis, Maria P, Vogels, Ronald, Brandenburg, Boerries, Kolkman, Joost A, Wilson, Ian A

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Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver von Zhang, Rui, El-Mayta, Rakan, Murdoch, Timothy J, Warzecha, Claude C, Billingsley, Margaret M, Shepherd, Sarah J, Gong, Ningqiang, Wang, Lili, Wilson, James M, Lee, Daeyeon, Mitchell, Michael J

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Intranasal gene therapy to prevent infection by SARS-CoV-2 variants von Sims, Joshua J, Greig, Jenny A, Michalson, Kristofer T, Lian, Sharon, Martino, R Alexander, Meggersee, Rosemary, Turner, Kevin B, Nambiar, Kalyani, Dyer, Cecilia, Hinderer, Christian, Horiuchi, Makoto, Yan, Hanying, Huang, Xin, Chen, Shu-Jen, Wilson, James M

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Extensive calcinosis cutis complicating radiotherapy von Ryan, Sarah, Kravvas, Georgios, Wilson, James M, Bunker, Christopher B

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CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX–knockout mice von Wang, Lili, Yang, Yang, Breton, Camilo Ayala, White, John, Zhang, Jia, Che, Yan, Saveliev, Alexei, McMenamin, Deirdre, He, Zhenning, Latshaw, Caitlin, Li, Mingyao, Wilson, James M.

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An Inherently Bifunctional Subset of Foxp3+ T Helper Cells Is Controlled by the Transcription Factor Eos von Sharma, Madhav D., Huang, Lei, Choi, Jeong-Hyeon, Lee, Eun-Joon, Wilson, James M., Lemos, Henrique, Pan, Fan, Blazar, Bruce R., Pardoll, Drew M., Mellor, Andrew L., Shi, Huidong, Munn, David H.

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ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing von Breton, Camilo, Clark, Peter M, Wang, Lili, Greig, Jenny A, Wilson, James M

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Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys von Hordeaux, Juliette, Hinderer, Christian, Buza, Elizabeth L, Louboutin, Jean-Pierre, Jahan, Tahsin, Bell, Peter, Chichester, Jessica A, Tarantal, Alice F, Wilson, James M

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Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol von Wang, Lili, Smith, Jeff, Breton, Camilo, Clark, Peter, Zhang, Jia, Ying, Lei, Che, Yan, Lape, Janel, Bell, Peter, Calcedo, Roberto, Buza, Elizabeth L, Saveliev, Alexei, Bartsevich, Victor V, He, Zhenning, White, John, Li, Mingyao, Jantz, Derek, Wilson, James M

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High activity of an affinity-matured ACE2 decoy against Omicron SARS-CoV-2 and pre-emergent coronaviruses von Sims, Joshua J, Lian, Sharon, Meggersee, Rosemary L, Kasimsetty, Aradhana, Wilson, James M

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Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals von Hinderer, Christian, Bell, Peter, Katz, Nathan, Vite, Charles H, Louboutin, Jean-Pierre, Bote, Erin, Yu, Hongwei, Zhu, Yanqing, Casal, Margret L, Bagel, Jessica, O'Donnell, Patricia, Wang, Ping, Haskins, Mark E, Goode, Tamara, Wilson, James M

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