Distortion Management in a Boundary Layer Ingestion Inlet Diffuser Using Hybrid Flow Control von Gissen, A. N, Vukasinovic, B, McMillan, M. L, Glezer, A

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Dynamics of flow control in an emulated boundary layer-ingesting offset diffuser von Gissen, A. N., Vukasinovic, B., Glezer, A.

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Flow Control of a Cascade Thrust Reverser von Gissen, A. N, Vukasinovic, B, Packard, N. O, Brzozowski, D. P, Glezer, 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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Epilepsy due to PNPO mutations: genotype, environment and treatment affect presentation and outcome von Mills, Philippa B., Camuzeaux, Stephane S.M., Footitt, Emma J., Mills, Kevin A., Gissen, Paul, Fisher, Laura, Das, Krishna B., Varadkar, Sophia M., Zuberi, Sameer, McWilliam, Robert, Stödberg, Tommy, Plecko, Barbara, Baumgartner, Matthias R., Maier, Oliver, Calvert, Sophie, Riney, Kate, Wolf, Nicole I., Livingston, John H., Bala, Pronab, Morel, Chantal F., Feillet, François, Raimondi, Francesco, Del Giudice, Ennio, Chong, W. Kling, Pitt, Matthew, Clayton, Peter T.

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Delivering efficient liver-directed AAV-mediated gene therapy von Baruteau, J, Waddington, S N, Alexander, I E, Gissen, P

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Whole exome sequencing identifies genetic variants in inherited thrombocytopenia with secondary qualitative function defects von Johnson, Ben, Lowe, Gillian C, Futterer, Jane, Lordkipanidzé, Marie, MacDonald, David, Simpson, Michael A, Sanchez-Guiú, Isabel, Drake, Sian, Bem, Danai, Leo, Vincenzo, Fletcher, Sarah J, Dawood, Ban, Rivera, José, Allsup, David, Biss, Tina, Bolton-Maggs, Paula Hb, Collins, Peter, Curry, Nicola, Grimley, Charlotte, James, Beki, Makris, Mike, Motwani, Jayashree, Pavord, Sue, Talks, Katherine, Thachil, Jecko, Wilde, Jonathan, Williams, Mike, Harrison, Paul, Gissen, Paul, Mundell, Stuart, Mumford, Andrew, Daly, Martina E, Watson, Steve P, Morgan, Neil V

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Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development von Massaro, Giulia, Geard, Amy F, Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N, Baruteau, Julien, Gissen, Paul, Rahim, Ahad A

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Comprehensive Rare Variant Analysis via Whole-Genome Sequencing to Determine the Molecular Pathology of Inherited Retinal Disease von Stephens, Jonathan, Dewhurst, Eleanor, Malka, Samantha, Plagnol, Vincent, Rizzo, Roberta, Scott, Richard H., Henderson, Robert H.H., MacLaren, Robert E., Paterson, Joan, Aitman, Timothy, Ali, Sonia, Ambegaonkar, Gautum, Arno, Gavin, Astle, William, Attwood, Antony, Bennett, David, Bitner-Glindzicz, Maria, Bleda, Marta, Boggard, Harm, Carss, Keren, Clements-Brod, Naomi, DaCosta, Rosa, De Vries, Minka, Dewhurst, Eleanor, Drewe, Elizabeth, Egner, William, Erber, Wendy N., Everington, Tamara, Fletcher, Debra, Freson, Kathleen, Gale, Daniel, Ghali, Neeti, Ghurye, Rohit, Gräf, Stefan, Greene, Daniel, Grigoriadou, Sofia, Grozeva, Detelina, Hackett, Scott, Hadinnapola, Charaka, Hague, Rosie, Hammerton, Tracey, Heemskerk, Johan W.M., Holder, Muriel, Holder, Susan, Huissoon, Aarnoud, Hurst, Jane, Jolles, Stephen, Keeling, David, Kennedy, Fiona, Kiely, David, Lawrie, Allan, Lear, Sara, Lees, Melissa, Lentaigne, Claire, Lorenzo, Lorena, Mangles, Sarah, Mapeta, Rutendo, Masati, Larahmie, Mathias, Mary, Michaelides, Michel, Millar, Carolyn M., Moledina, Shahin, Moore, Anthony, Murng, Sai, Oksenhendler, Eric, Park, Soo-Mi, Patch, Chris, Paterson, Joan, Penkett, Christopher J., Pepke-Zaba, Joanna, Pollock, Val, Qasim, Waseem, Quinti, Isabella, Reid, Evan, Rondina, Matthew, Rosser, Elisabeth, Santra, Saikat, Sargur, Ravishankar, Savic, Sinisa, Scully, Marie, Sewell, Carrock, Smith, Kenneth, Southgate, Laura, Stauss, Hans, Stein, Penelope, Talks, Kate, Thomas, Ellen, Thrasher, Adrian, Tischkowitz, Marc, Titterton, Catherine, Turro, Ernest, von Ziegenweldt, Julie, Vonk Noordegraaf, Anton, Wakeling, Emma, Wanjiku, Ivy, Welch, Steve, Westbury, Sarah, Woods, Geoffrey, Yong, Patrick, Webster, Andrew R.

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Use of next‐generation sequencing and candidate gene analysis to identify underlying defects in patients with inherited platelet function disorders von Leo, V. C., Morgan, N. V., Bem, D., Jones, M. L., Lowe, G. C., Lordkipanidzé, M., Drake, S., Simpson, M. A., Gissen, P., Mumford, A., Watson, S. P., Daly, M. E.

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Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer von Baruteau, Julien, Perocheau, Dany P., Hanley, Joanna, Lorvellec, Maëlle, Rocha-Ferreira, Eridan, Karda, Rajvinder, Ng, Joanne, Suff, Natalie, Diaz, Juan Antinao, Rahim, Ahad A., Hughes, Michael P., Banushi, Blerida, Prunty, Helen, Hristova, Mariya, Ridout, Deborah A., Virasami, Alex, Heales, Simon, Howe, Stewen J., Buckley, Suzanne M. K., Mills, Philippa B., Gissen, Paul, Waddington, Simon N.

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Vps33b is crucial for structural and functional hepatocyte polarity von Hanley, Joanna, Dhar, Dipok Kumar, Mazzacuva, Francesca, Fiadeiro, Rebeca, Burden, Jemima J, Lyne, Anne-Marie, Smith, Holly, Straatman-Iwanowska, Anna, Banushi, Blerida, Virasami, Alex, Mills, Kevin, Lemaigre, Frédéric P, Knisely, A.S, Howe, Steven, Sebire, Neil, Waddington, Simon N, Paulusma, Coen C, Clayton, Peter, Gissen, Paul

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Expanding the phenotype in argininosuccinic aciduria: need for new therapies von Baruteau, Julien, Jameson, Elisabeth, Morris, Andrew A., Chakrapani, Anupam, Santra, Saikat, Vijay, Suresh, Kocadag, Huriye, Beesley, Clare E., Grunewald, Stephanie, Murphy, Elaine, Cleary, Maureen, Mundy, Helen, Abulhoul, Lara, Broomfield, Alexander, Lachmann, Robin, Rahman, Yusof, Robinson, Peter H., MacPherson, Lesley, Foster, Katharine, Chong, W. Kling, Ridout, Deborah A., Bounford, Kirsten McKay, Waddington, Simon N., Mills, Philippa B., Gissen, Paul, Davison, James E.

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Regulation of post-Golgi LH3 trafficking is essential for collagen homeostasis von Banushi, Blerida, Forneris, Federico, Straatman-Iwanowska, Anna, Strange, Adam, Lyne, Anne-Marie, Rogerson, Clare, Burden, Jemima J., Heywood, Wendy E., Hanley, Joanna, Doykov, Ivan, Straatman, Kornelis R., Smith, Holly, Bem, Danai, Kriston-Vizi, Janos, Ariceta, Gema, Risteli, Maija, Wang, Chunguang, Ardill, Rosalyn E., Zaniew, Marcin, Latka-Grot, Julita, Waddington, Simon N., Howe, S. J., Ferraro, Francesco, Gjinovci, Asllan, Lawrence, Scott, Marsh, Mark, Girolami, Mark, Bozec, Laurent, Mills, Kevin, Gissen, Paul

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Phenotypic spectrum of neurodegeneration associated with mutations in the PLA2G6 gene (PLAN) von KURIAN, M. A, MORGAN, N. V, ROSSER, E. M, WASSMER, E, GISSEN, P, MAHER, E. R, MACPHERSON, L, FOSTER, K, PEAKE, D, GUPTA, R, PHILIP, S. G, HENDRIKSZ, C, MORTON, J. E. V, KINGSTON, H. M

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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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Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort von Perocheau, Dany P, Cunningham, Sharon, Lee, Juhee, Antinao Diaz, Juan, Waddington, Simon N, Gilmour, Kimberly, Eaglestone, Simon, Lisowski, Leszek, Thrasher, Adrian J, Alexander, Ian E, Gissen, Paul, Baruteau, Julien

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Cerebrospinal fluid neurofilament light chain levels in CLN2 disease patients treated with enzyme replacement therapy normalise after two years on treatment [version 2; peer review... von Iwan, Katharina, Patel, Nina, Heslegrave, Amanda, Borisova, Mina, Lee, Laura, Bower, Rebecca, Mole, Sara E, Mills, Philippa B, Zetterberg, Henrik, Mills, Kevin, Gissen, Paul, Heywood, Wendy E

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Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice von Touramanidou, Loukia, Gurung, Sonam, Cozmescu, Claudiu A, Perocheau, Dany, Moulding, Dale, Finn, Patrick F, Frassetto, Andrea, Waddington, Simon N, Gissen, Paul, Baruteau, Julien

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Cathepsin D deficiency causes juvenile-onset ataxia and distinctive muscle pathology von Hersheson, Joshua, Burke, Derek, Clayton, Robert, Anderson, Glenn, Jacques, Thomas S, Mills, Philippa, Wood, Nicholas W, Gissen, Paul, Clayton, Peter, Fearnley, Julian, Mole, Sara E, Houlden, Henry

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