121 A Retrospective, Non-inferiority Treat-at-Home Study Utilizing a Surfactant-based Dressing for Partial-thickness Burn Wounds (HOME) von Lyle, Elysha, Jarreau, Jacob T, Sanders, Matthew, Jones, Maria Tucci, Knight, Denise, Brockway, Patrick, Hassouba, Mahmoud, Velamuri, Sai R, Hill, David M

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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome von Gentner, Bernhard, Tucci, Francesca, Galimberti, Stefania, Fumagalli, Francesca, De Pellegrin, Maurizio, Silvani, Paolo, Camesasca, Chiara, Pontesilli, Silvia, Darin, Silvia, Ciotti, Francesca, Sarzana, Marina, Consiglieri, Giulia, Filisetti, Chiara, Forni, Giulia, Passerini, Laura, Tomasoni, Daniela, Cesana, Daniela, Calabria, Andrea, Spinozzi, Giulio, Cicalese, Maria-Pia, Calbi, Valeria, Migliavacca, Maddalena, Barzaghi, Federica, Ferrua, Francesca, Gallo, Vera, Miglietta, Simona, Zonari, Erika, Cheruku, Patali S, Forni, Claudia, Facchini, Marcella, Corti, Ambra, Gabaldo, Michela, Zancan, Stefano, Gasperini, Serena, Rovelli, Attilio, Boelens, Jaap-Jan, Jones, Simon A, Wynn, Robert, Baldoli, Cristina, Montini, Eugenio, Gregori, Silvia, Ciceri, Fabio, Valsecchi, Maria G, la Marca, Giancarlo, Parini, Rossella, Naldini, Luigi, Aiuti, Alessandro, Bernardo, Maria-Ester

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Prevalence and impact of COVID-19 sequelae on treatment and survival of patients with cancer who recovered from SARS-CoV-2 infection: evidence from the OnCovid retrospective, multi... von Bower, Mark, Patel, Meera, Dolly, Saoirse, Loizidou, Angela, Chester, John, Mukherjee, Uma, Dalla Pria, Alessia, Ottaviani, Diego, Chowdhury, Amani, Merry, Eve, Salazar, Ramon, Bertuzzi, Alexia, Brunet, Joan, Tagliamento, Marco, Pous, Anna, Sita-Lumsden, Ailsa, Srikandarajah, Krishnie, Colomba, Johann, Pommeret, Fanny, Seguí, Elia, Generali, Daniele, Grisanti, Salvatore, Pedrazzoli, Paolo, Rizzo, Gianpiero, Moss, Charlotte, Evans, Joanne S, Harbeck, Nadia, Biello, Federica, Bertulli, Rossella, Liñan, Raquel, Rossi, Sabrina, Tondini, Carlo, Fox, Laura, Baggi, Alice, Fotia, Vittoria, Parisi, Alessandro, Porzio, Giampero, Saponara, Maristella, Cruz, Claudia Andrea, García-Illescas, David, Felip, Eudald, Sharkey, Rachel, Roldán, Elisa, Reyes, Roxana, Earnshaw, Irina, Ferrante, Daniela, Marco-Hernández, Javier, Ruiz-Camps, Isabel, Patriarca, Andrea, Sureda, Anna, Martinez-Vila, Clara, Sanchez de Torre, Ana, Cantini, Luca, Filetti, Marco, Rimassa, Lorenza, Chiudinelli, Lorenzo, Franchi, Michela, Santoro, Armando, Prat, Aleix, Diamantis, Nikolaos, Newsom-Davis, Thomas, Gennari, Alessandra, Cortellini, Alessio, Swallow, Judith, Chopra, Neha, Sng, Christopher CT, Wong, Yien Ning Sophia, Benafif, Sarah, Dileo, Palma, Wu, Anjui, Sinclair, Alasdair, Soosaipillai, Gehan, Jones, Eleanor, Diamantis, Nikolaos, Mukherjee, Uma, Jackson, Amanda, Colomba, Johann, Colomba-Blameble, Emeline, Prat, Aleix, Reyes, Roxana, Segui, Elia, Aguilar-Company, Juan, Ruiz-Camps, Isabel, Garcia Illescas, David, Mirallas, Oriol, Roldán, Elisa, Carbó, Anna, Mesia, Ricard, Maluquer, Clara, D'Avanzo, Francesca, Tonini, Giuseppe, Provenzano, Salvatore, Tovazzi, Valeria, Queirolo, Paola, Mazzoni, Francesca, Tucci, Marco, Berardi, Rossana, Guida, Annalisa, Bracarda, Sergio, Iglesias, Maria

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SARS-CoV-2 omicron (B.1.1.529)-related COVID-19 sequelae in vaccinated and unvaccinated patients with cancer: results from the OnCovid registry von Mukherjee, Uma, Salazar, Ramon, Sureda, Anna, Ferrante, Daniela, Bower, Mark, Sharkey, Rachel, Mirallas, Oriol, Plaja, Andrea, Cucurull, Marc, Mesia, Ricard, Dalla Pria, Alessia, Newsom-Davis, Thomas, Van Hemelrijck, Mieke, Sita-Lumsden, Ailsa, Apthorp, Eleanor, Vincenzi, Bruno, Di Fazio, Giuseppina Rita, Tonini, Giuseppe, Pantano, Francesco, Bertuzzi, Alexia, Brunet, Joan, Lambertini, Matteo, Pedrazzoli, Paolo, Biello, Federica, D'Avanzo, Francesca, Lee, Alvin J X, Shawe-Taylor, Marianne, Rogers, Lucy, Murphy, Cian, Cooper, Lee, Andaleeb, Ramis, Khalique, Saira, Bawany, Samira, Carmona-García, M Carmen, Fort-Culillas, Roser, Liñan, Raquel, Zoratto, Federica, Rizzo, Gianpiero, Perachino, Marta, Doonga, Kris, Gaidano, Gianluca, Pérez Criado, Ignacio, Giusti, Raffaele, Mazzoni, Francesca, Antonuzzo, Lorenzo, Parisi, Alessandro, Aujayeb, Avinash, Rimassa, Lorenza, Diamantis, Nikolaos, Bertulli, Rossella, Fulgenzi, Claudia A M, D'Alessio, Antonio, Saoudi-Gonzalez, Nadia, Garcia Illescas, David, Medina, Irene, Fox, Laura, Gennari, Alessandra, Pinato, David J, Evans, Joanne S, Hanbury, Georgina, Chung, Chris, Patel, Meera, Belessiotis, Katherine, Saorise, Dolly, Jones, Eleanor, Apthorp, Eleanor, Russell, Beth, Townsend, Sarah, Jackson, Amanda, Loizidou, Angela, Piccart, Martine, Pommeret, Fanny, Cruz, Claudia A, Marco-Hernández, Javier, Viladot, Margarita, Harbeck, Nadia, Wuerstlein, Rachel, Henze, Franziska, Mahner, Sven, Felip, Eudald, Scotti, Lorenza, Marrari, Andrea, Fusco, Vittorio, Delfanti, Sara, Zambelli, Alberto, Franchi, Michela, Libertini, Michela, Generali, Daniele, Grisanti, Salvatore, Baggi, Alice, Tovazzi, Valeria, Ficorella, Corrado, Saponara, Maristella, Filetti, Marco, Tucci, Marco, Berardi, Rossana, Paoloni, Francesco, Bracarda, Sergio, Iglesias, Maria, Sanchez de Torre, Ana

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Outcomes from elective colorectal cancer surgery during the SARS‐CoV‐2 pandemic von Chand, Manish, Di Saverio, Salomone, Gallo, Gaetano, Adamina, Michel, Blanco‐Colino, Ruth, Breen, Kerry A., Burke, Joshua, Drake, Thomas M., Gallo, Gaetano, Griffiths, Ewen A., Harrison, Ewen M., Mohan, Helen M., Ntirenganya, Faustin, Rivera, Jose Perez Carlos, Schache, Andrew, Tsoulfas, Georgios, Egoroff, N., Bong, C., Dudi‐Venkata, N. N., Grechenig, M., Gill, R., Stevenson, J., Skrovina, M., Lakkis, Z., Denet, C., Kalfountzos, C., Parray, F., Mohammad, Z., Corless, K., Marino, M. V., Bellora, P., Rossi, S., Montori, G., Aversano, A., Delrio, P., De Simone, V., Capelli, P., Mochet, S., Laface, L., Sapienza, P., Norcini, C., Neri, J., Palagi, S., Agresta, F., Gallo, G., Obeidat, K., Rasoaherinomenjanahary, F., Oosterling, S., Ramesh, P., Costa, M. J. M. A., Nunes Coelho, M., Ribeiro, R., Gomes, J., Vieira Paiva Lopes, A. C., Ceranic, M., Jelenkovic, J., Goran, M., Valle Rubio, A., Otero, A., Moro‐Valdezate, D., Peña, E., Picón Rodríguez, R., Saudí, S., Pacheco Sánchez, D., Abad‐Motos, A., Heras Aznar, J., Jayarajah, U., Löfgren, N., Mongelli, F., Guglielmetti, L., Hamzaoglu, I., Velidedeoglu, M., Mantoglu, B., Tanal, M., Kaya, T., Tugmen, C., Singh, A., Townson, A., Kler, A., Emerson, H., Parwaiz, I., Boulton, A. J., Velchuru, V., Johnstone, J., Kaul, S., Williams, G., Thaha, M., McAleer, J., Lyons, A., Hainsworth, A., Watson, L. J., Kapoor, S., Chand, M., Baig, M., Shortland, T., Seymour, Z., Tsai, L., Sarin, A., Foley, E., Fahey, B. A.

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P037: Design of a multi-center randomized phase 3 clinical trial (HURCULES) evaluating OTL-203 in MPS-IH vs allogeneic hematopoietic stem cell transplantation von Harmatz, Paul, Wynn, Robert, Gupta, Ashish, Kharbanda, Sandhya, Lindemans, Caroline, Ahrens-Nicklas, Rebecca, van Hasselt, Peter, Lund, Troy, Olson, Timothy, Tucci, Francesca, Martin, Leonie, Boeglin, Nathalie, Brooks, Jean, Syonmez, Su, Campbell, Laura, Jones, Simon, Orchard, Paul, Bernardo, Maria Ester

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Design of a multi-center randomized active controlled phase 3 clinical trial (HURCULES) evaluating the safety and efficacy of OTL-203 in patients with MPS IH versus standard of car... von Orchard, Paul J., Wynn, Robert F., Kharbanda, Sandhya, Ahrens-Nicklas, Rebecca, van Hasselt, Peter M., Lund, Troy C., Olson, Timothy S., Tucci, Francesca, Martin, Leonie, Boeglin, Nathalie, Brooks, Jean, Syonmez, Su, Campbell, Laura, Harmatz, Paul, Jones, Simon A., Gupta, Ashish O., Bernardo, Maria Ester

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DYNC2H1 hypomorphic or retina-predominant variants cause nonsyndromic retinal degeneration von Vig, Anjali, Poulter, James A., Ottaviani, Daniele, Tavares, Erika, Toropova, Katerina, Tracewska, Anna Maria, Mollica, Antonio, Kang, Jasmine, Kehelwathugoda, Oshini, Paton, Tara, Maynes, Jason T., Wheway, Gabrielle, Arno, Gavin, Khan, Kamron N., McKibbin, Martin, Toomes, Carmel, Ali, Manir, Di Scipio, Matteo, Li, Shuning, Ellingford, Jamie, Black, Graeme, Webster, Andrew, Rydzanicz, Małgorzata, Stawiński, Piotr, Płoski, Rafał, Vincent, Ajoy, Cheetham, Michael E., Inglehearn, Chris F., Roberts, Anthony, Heon, Elise

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The inner junction protein CFAP20 functions in motile and non-motile cilia and is critical for vision von Chrystal, Paul W., Lambacher, Nils J., Doucette, Lance P., Bellingham, James, Schiff, Elena R., Noel, Nicole C. L., Li, Chunmei, Tsiropoulou, Sofia, Casey, Geoffrey A., Zhai, Yi, Nadolski, Nathan J., Majumder, Mohammed H., Tagoe, Julia, D’Esposito, Fabiana, Cordeiro, Maria Francesca, Downes, Susan, Clayton-Smith, Jill, Ellingford, Jamie, Mahroo, Omar A., Hocking, Jennifer C., Cheetham, Michael E., Webster, Andrew R., Jansen, Gert, Blacque, Oliver E., Allison, W. Ted, Au, Ping Yee Billie, MacDonald, Ian M., Arno, Gavin, Leroux, Michel R.

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Biallelic variants in PCDHGC4 cause a novel neurodevelopmental syndrome with progressive microcephaly, seizures, and joint anomalies von Iqbal, Maria, Maroofian, Reza, Çavdarlı, Büşranur, Riccardi, Florence, Field, Michael, Banka, Siddharth, Bubshait, Dalal K., Li, Yun, Hertecant, Jozef, Baig, Shahid Mahmood, Dyment, David, Efthymiou, Stephanie, Abdullah, Uzma, Makhdoom, Ehtisham Ul Haq, Ali, Zafar, Scherf de Almeida, Tobias, Molinari, Florence, Mignon-Ravix, Cécile, Chabrol, Brigitte, Antony, Jayne, Ades, Lesley, Pagnamenta, Alistair T., Jackson, Adam, Douzgou, Sofia, Beetz, Christian, Karageorgou, Vasiliki, Vona, Barbara, Rad, Aboulfazl, Baig, Jamshaid Mahmood, Sultan, Tipu, Alvi, Javeria Raza, Maqbool, Shazia, Rahman, Fatima, Toosi, Mehran Beiraghi, Ashrafzadeh, Farah, Imannezhad, Shima, Karimiani, Ehsan Ghayoor, Sarwar, Yasra, Khan, Sheraz, Jameel, Muhammad, Noegel, Angelika A., Budde, Birgit, Altmüller, Janine, Motameny, Susanne, Höhne, Wolfgang, Houlden, Henry, Nürnberg, Peter, Wollnik, Bernd, Villard, Laurent, Alkuraya, Fowzan Sami, Osmond, Matthew, Hussain, Muhammad Sajid, Yigit, Gökhan

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Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010 von Ferrua, Francesca, Cicalese, Maria Pia, Galimberti, Stefania, Giannelli, Stefania, Dionisio, Francesca, Barzaghi, Federica, Migliavacca, Maddalena, Bernardo, Maria Ester, Calbi, Valeria, Tucci, Francesca, Assanelli, Andrea A., Peccatori, Jacopo, Albertazzi, Elena, Clerici, Alessandra G., Salerio, Federica A., Scala, Serena, Basso-Ricci, Luca, Cenciarelli, Sabina, Canarutto, Daniele, Fraschetta, Federico, Bartoli, Antonella, Wolf, Hermann M., Silvani, Paolo, Gattillo, Salvatore, Coppola, Milena, Santoleri, Luca, Villa, Anna, Jones, Russell, Dott, Chris, Grazia Valsecchi, Maria, Ciceri, Fabio, Naldini, Luigi, Aiuti, Alessandro

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Design of a Multi-Center Randomized Active Controlled Phase 3 Clinical Trial (HURCULES) Evaluating the Safety and Efficacy of OTL-203 in Patients with MPS-IH Versus Standard of Car... von Gupta, Ashish O., Wynn, Robert F, Kharbanda, Sandhya, Lindemans, Caroline A., Ahrens-Nicklas, Rebecca, van Hasselt, Peter M., Lund, Troy C., Olson, Timothy S., Tucci, Francesca, Martin, Leonie, Boeglin, Nathalie, Brooks, Jean, Syonmez, Su, Campbell, Laura, Harmatz, Paul, Jones, Simon A., Orchard, Paul J., Bernardo, Maria Ester

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Constructing custom-made radiotranscriptomic signatures of vascular inflammation from routine CT angiograms: a prospective outcomes validation study in COVID-19 von Xie, Cheng, Alexander, Donna, O’Connor, Daniel, Lumley, Sheila F, Constantinides, Bede, Chau, Kevin K, Lodge, Archie, Indrajeet, Das, Deshpande, Aparna, Bajaj, Amrita, Farid, Shakil, Krasopoulos, George, Sayeed, Rana, Neubauer, Stefan, Channon, Keith M, Antoniades, Charalambos, Ahern, David J, Arancibia-Cárcamo, Carolina, Brent, Andrew, Burnham, Katie, Coles, Mark, Cribbs, Adam, Dib, Lea, Dold, Christina, Dunachie, Susanna, Espinosa, Alexis, Fries, Anastasia, Godfrey, Leila, Gomez Vazquez, Maria, Heilig, Raphael, Hinds, Charles, Hird, Clare, Hoekzema, Renee, Hughes, Jim, Hutton, Paula, Jackson-Wood, Matthew, James-Bott, Anna, Jones, Elizabeth, Kurupati, Prathiba, Kwok, Andrew, Lonie, Lorne, Marsden, Brian, Martinez, Jose, Mazurczyk, Michalina, McGowan, Simon, Mentzer, Alexander, Mi, Yuxin, O'Brien, Darragh, Ogg, Graham, Peng, Yanchun, Psaila, Bethan, Repapi, Emmanouela, Silva-Reyes, Laura, Richard, Jean-Baptiste, Ruehle, Fabian, Sansom, Stephen Nicholas, Schwessinger, Ron, Slawinski, Hubert, Sobrinodiaz, Alberto, Stafford, Lizzie, Stockdale, Lisa, Strickland, Marie, Taylor, Chelsea, Tong, Orion, Udalova, Irina, Uhlig, Holm, Wang, Lihui, Watkinson, Peter, Wray, Katherine, Youngs, Jonathan, Banning, Adrian, Koo, Bon-Kwon, Shirodaria, Cheerag, Adlam, David, Newby, David, Connolly, Derek, Alexander, Donna, McAlindon, Elisa, Pugliese, Francesca, He, Guo-Wei, Kondo, Hidekazu, Benedek, Imre, Graby, John, Fan, Li-Juan, Marwan, Mohamed, Takahashi, Naohiko, Mehta, Nehal, Dai, Neng, Screaton, Nicholas, Kotronias, Rafail, Kharbanda, Rajesh, Preston, Rebecca, Mirsadraee, Saeed, Thomas, Sheena, Klömpken, Steffen, Petersen, Steffen, Achenbach, Stephan, Benedek, Theodora, Sharma, Vinoda, Lin, Wen-Hua

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Biallelic variants in coenzyme Q10 biosynthesis pathway genes cause a retinitis pigmentosa phenotype von Jurkute, Neringa, Cancellieri, Francesca, Pohl, Lisa, Li, Catherina H. Z., Heaton, Robert A., Reurink, Janine, Bellingham, James, Quinodoz, Mathieu, Yioti, Georgia, Stefaniotou, Maria, Weener, Marianna, Zuleger, Theresia, Haack, Tobias B., Stingl, Katarina, Hoyng, Carel B., Mahroo, Omar A., Hargreaves, Iain, Raymond, F. Lucy, Michaelides, Michel, Rivolta, Carlo, Kohl, Susanne, Roosing, Susanne, Webster, Andrew R., Arno, Gavin

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European DataGrid Project: Experiences of Deploying a Large Scale Testbed for E-science Applications von Gagliardi, Fabrizio, Jones, Bob, Reale, Mario, Burke, Stephen

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