Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates

Autor: Helena Costa-Verdera, Fanny Collaud, Christopher R. Riling, Pauline Sellier, Jayme M. L. Nordin, G. Michael Preston, Umut Cagin, Julien Fabregue, Simon Barral, Maryse Moya-Nilges, Jacomina Krijnse-Locker, Laetitia van Wittenberghe, Natalie Daniele, Bernard Gjata, Jeremie Cosette, Catalina Abad, Marcelo Simon-Sola, Severine Charles, Mathew Li, Marco Crosariol, Tom Antrilli, William J. Quinn, David A. Gross, Olivier Boyer, Xavier M. Anguela, Sean M. Armour, Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi

Publikováno v: Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)

Pompe disease is currently treated with enzyme replacement therapy (ERT) with recombinant human acid alpha-glucosidase (GAA). Here, the authors show hepatic-directed gene therapy with AAV vectors enhances GAA bioavailability compared with ERT, result

Externí odkaz: https://doaj.org/article/ecdd7a66d5fe4b0e825ff6e616f835cf

Guidance on mucositis assessment from the MASCC Mucositis Study Group and ISOO: an international Delphi studyResearch in context

Autor: Ragda Abdalla-Aslan, Pierluigi Bonomo, Dorothy Keefe, Nicole Blijlevens, Katrina Cao, Yin Ting Cheung, Eduardo Rodrigues Fregnani, Robert Miller, Judith Raber-Durlacher, Joel Epstein, Ysabella Van Sebille, Elisa Kauark-Fontes, Abhishek Kandwal, Emma McCurdy-Franks, Joel Finkelstein, Victoria McCarvell, Yehuda Zadik, Giulia Ottaviani, Rui Amaral Mendes, Caroline Margina Speksnijder, Hannah Rose Wardill, Paolo Bossi, Alexa Laheij, Arghavan Tonkaboni, Jacqui Scott, Rania Abasaeed, Adel Kauzman, Adriana Do Socorro Lima Flato, Adwaita Gore, Anne-Marie Hardman, Agnes Horvath, Allan Hovan, Aisha Al-Jamaei, Aya koizumi, Alan Santos-Silva, Alessandra Majorana, Alexandre Giannini, Aléxia Teixeira, Muhammad Ali Shazib, Alison Melvin, Aluísio Miranda Filho, Amanda Gruza, Amber Brown-Dahl, Amit Harilall, Amr El Maghrabi, Ana Andabak Rogulj, Ana Raphaela Curvo, Ana Laura Soares, Andrea Stringer, Andréa Moreira, Andy Kurtzweil, Angelyn Salaberry, Anne Blazy, Anne Margrete Gussgard, Anne Marie Lynge Pedersen, Annette Freidank, Anura Ariyawardana, Adrian Ramseier, Jann Arends, Ariel Blanchard, Adriana Sesti Paz, Angela Thermann, Augusto Poropat, Azael Freites-Martinez, Abdul Rahman Al-Azri, Bente Brokstad Herlofson, Sitaraman BalajiSubramanian, Barbara Ballantyne, Kivanc Bektas-Kayhan, Bengt Hasséus, Benjamin Kaffenberger, Bernar Benites, Bernice Kwong, Beth Test, Fernando Chiantia, Bo Pettersson, Bomi Framroze, Božana Lončar Brzak, Brittany Dulmage, Sorin Buga, Caroline Spekssnijder, Carlton Brown, Antonio Carlos Moura de Melo, Ana Carolina Ribeiro, Caroline Silva, Caroline Fulop, Carryn Anderson, Catherine Flaitz, Cathy Massoud, Cesar Migliorati, Callie Gross, Chiara Gandini, Charles Loprinzi, Charlotte de Mooij, Catherine Hong, Ying Chu Choi, Maria Choy, Christine Boers-Doets, Leonard Schmeel, Cibele Nagano, Maria Coeli Franco, Courtney Subramaniam, Carolyn Patrick, Catherine Poh, Cristina Neuenschwander, Cesar Virgen, Dorothea Riesenbeck, Dale Weaver, Daniel Cohen Goldemberg, Daniel Sundaresan, Daniela Nunes, Danyel Perez, Daphine Travassos, David Yang, Daniela Ribeiro, Dean Kolbinson, Deborah Buick, Deborah Saunders, Juliane De Bortolli, Deepika Chugh, Denise Markstrom, Denise Travassos, Dianna Weikel, Dimitra Galiti, Dinusha Peiris, Fedja Djordjevic, Pankaj Singhai, Douglas Peterson, Douglas Fonseca, Doreen Pon, Iuliia Kovalenko, Aleksandra Polonskaia, Rogério Caldas, Kevin Saganski, Julia Néri, Dennis Abbott, Abhijna Vithal Yergolkar, Cristina Del Conte, Januaria Passos, Katia Uezu, Paula Silva, Steven Gilbert, Keng Yeoh, Kunal Jain, Madhup Rastogi, Satheeshkumar Poolakkad Sankaran, Deborah Manne, Evgeniya Shatokhina, Esther Adebayo-Olojo, Eszter Somogyi-Ganss, Eli Ehrenpreis, Wilber Bernaola-Paredes, Eduardo Fregnani, Elaci Cardoso, Elena Bardellini, Eleni Arvanitou, Elisa Kauark Fontes, Elise Bruning, Eloise Neumann, Elsa Madureira, Marcia Ramires, Erofili Papadopoulou, Etiene Munhoz, Fred Spijkervet, Fabiana Granzotto, Fabiana Martins, Fabio Alves, Farah Mougeot, Federica Aielli, Fernanda Pigatti, Fernanda Fonseca, Firoozeh Samim, Flavia Carvalho, Florence Cuadra Zelaya, Cesar Freytes, Gabriela da Silveira, Gabriela Torino, Gabriela Martins, Geisa Silva, Gemma Caro, Gemma Bryan, Georgette Radford, Ghanyah Al-Qadami, Giorgia Albini, Gisele Mainville, Georgios Gkardiakos, Gleidston Potter, Gulbin Hoeberechts, Gordon Howarth, Grace Bradley, Gunjan Verma, Gustavo dos Santos, Margaret Randles-Guzzardi, Hanlie Engelbrecht, Hannah Wardill, Heidi Hansen, Iquebal Hasan, Hironobu Hata, Helena Ullgren, Heliton Spindola Antunes, Heloísa Laís dos Santos, Howard Weld, Helen McInnes, Hans Peter Jungbluth, Hsiaofen Weng, Ian Hewson, Ingrid Santos, Jorge Illarramendi, Ines Semendric, Rol Menge, Inger Von Bultzingslowen, Maria da Gloria de Melo, Iona Leong, Isabella Fonseca, Isadora Kalif, James Carroll, Janet Coller, Johann Beck-Mannagetta, Joanne Bowen, Jose Meurer, Ricky McCullough, Jennifer Powers, Jesus Gomez, Jimma Lenjisa, jaya Vangara, Jasna Leko, Jane Fall-Dickson, Jean-Luc Mougeot, Joan Fox, Jolien Robijns, Jonn Wu, Patricio Palma, Jaya Amaram-Davila, Jim Siderov, Juliana Dantas, Juliana Jasper, Juliana Monteiro, Julia Bruno, julie pfeffer, Julija Jovanovic Ristivojevic, Juliana Brito, Jyothsna Kuriakose, Yuji Kabasawa, Kanan Dave, Karin Barczyszyn, Karol Sartori Lima, Kate Secombe, Kate White, Kate Cooper, Kouji Katsura, Karen Biggs, Katharine Ciarrocca, Kristopher Dennis, Ken Tomizuka, Kevin Hendler, Ikuko Komo, Kristina Skallsjö, Kristy Hodgins, Katia Rupel, Keiko Tanaka, Seema Kurup, Luiz Gueiros, Larissa Agatti, Laura Garzona-Navas, Letícia Guerra, Leila Portela, Leilani Iossi, Linda Elting, Lene Baad-Hansen, Leslie Reeder, Leticia Lang, Liciane Menezes, Liliana Braun, Liliane Grando, Mathew Lim, Lina Fernandez, Lucy McKeage, Luana Campos, Luciana Simonato, Luciana Muniz, Leah van Draanen, Mieko Mizutani, Tsai-Wei Huang, Mahfujul Riad, Mahnoor Nazar, Maíra Souza, Mariana Minamisako, Manoela Pereira, Carlos Mantelato, Márcio Diniz-Freitas, Marco Montezuma, Marco Andrade, Marcos Santos, Margherita Gobbo, Maria Caterina Fortuna, Mariana Vitor, Joana Marinho, Alina Markova, Marlyse Knuchel, Marta Carlesimo, Marta Neves, Andrew Mazar, Maria Cristina Gomez Amarilla, Mark Chambers, Melissa de Araujo, Alexandre Melo, Melody Cole, Mohamed Elsayed, Monica Fliedner, Martin Hauer-Jensen, Micaela Bouchacourt, Michael Brennan, Michael Thirlwell, Michio Nakamura, Midori Nakagaki, Camila Rossi, Mireille Kaprilian, Michael Kase, Michael Dougan, Monique Stokman, Ragnhild Monsen, Alisha Morgan, Jocelyn Harding, Maryam Taleghani, Marie-Therese Genot, Mukund Seshadri, Brian Muzyka, Nancy Batista, Nancy Gadd, Naoko Tanda, Narmin Nasr, Natália Garcia, Nathan Lee, Natalia Palmier, Norman Brito-Dellan, Nancy Corbitt, Neli Pieralisi, Verônica Serrano, Nicola Alessandro Iacovelli, Norma Lúcia Sampaio, Nour Karra, Niveditha Venkatesh, Noam Yarom, Renata Cristina Borin, Olivia Lemenchick, Ondina Mendes, Ourania Nicolatou-Galitis, Vasiliy Shchitka, Paula Reis, Paulo Sérgio Santos, Paz Fernandez-Ortega, Ira Parker, Raquel García, Peter Fritz, Edmund Peters, Pamela Gardner, Pierre Saint Girons, Priya Tiwari, Pravin Chaturvedi, Tais de Moraes, Priscila Andrade, Raj Nair, Rachel Gibson, Rachita Gururaj, Raghu Thota, Rajesh Lalla, Raquel Almeida Prado, Ravikiran Ongole, George Taybos, Regina Mackey, Renata Rego, Renata Camilotti, Renata Ferrari, Renato Junior, Rene-Jean Bensadoun, Richard Logan, Roberta Sales, Roberta Zanicotti, Roberta Tunes, Rodolfo Mauceri, Rosiene Feitoza, Kathryn Ruddy, Cynthia Rybczyk, Stephanie Trager, Sachiyo Mitsunaga, Sahani Gunathilake, Rajan Saini, Viola Salvestrini, Sandip Mukhopadhyay, Sandrina Angeloz, Pramod Sankar S, Luciana S Barbosa Barbosa, Elena Volkova, Sharon Elad, Sergio Cantoreggi, Sharon Gordon, Shelly Brown, Shu Yie Janine Tam, Sibelle Faleiro, Silmara da Silva, Silvia de Oliveira, Siri Beier Jensen, Ivana Skrinjar, Sophie Beaumont, Felipe Sperandio, Sandra Reese, Steven Roser, Sachiko Seo, Stephanie van Leeuwen, Stephen Sonis, Stephen Bernard, Stephen Rajan Samuel, Stuart Taylor, Suranjan Maitra, Susanne Skulski, Suzanne Carlisle, Sylvie Louise Avon, Tomoya Yokota, Takashi Yurikusa, Tabata Santos Polvora, Tabitha Kelock, Tauana Fernandes, Taylor Wain, Timothy Brown, Tetsuhito konishi, Thalyta Amanda Ferreira, Tomoko Kataoka, Thomas Kelly, Takehiko Mori, Tomoko Higuchi, Toshiaki Saeki, Nikolaos Tsoukalas, Typhaine Maupoint De Vandeul, Masatoshi Usubuchi, Vanessa Lacerda, Vanessa Tilly, Emmanuelle Vigarios, Alessandro Villa, Vinicius Torregrossa, Vinodh Kumar Selvaraj, Viviane Sarmento, Vivien Heng, Wagner Gomes-Silva, Petter Wilberg, Wanessa Miranda e Silva, Wan Nor I'zzah Wan Mohamad Zain, Wonse Park, Wim Tissing, Yoshihiko Soga, Bella Van Sebille, Yuhei Matsuda

Publikováno v: EClinicalMedicine, Vol 73, Iss , Pp 102675- (2024)

Summary: Background: Mucositis is a common and highly impactful side effect of conventional and emerging cancer therapy and thus the subject of intense investigation. Although common practice, mucositis assessment is heterogeneously adopted and poorl

Externí odkaz: https://doaj.org/article/057c9a97cdb74c5aa6da891b8ee0c0af

People do change their beliefs about conspiracy theories—but not often

Autor: Matt N. Williams, Mathew Ling, John R. Kerr, Stephen R. Hill, Mathew D. Marques, Hollie Mawson, Edward J. R. Clarke

Publikováno v: Scientific Reports, Vol 14, Iss 1, Pp 1-10 (2024)

Abstract Recent research has produced a significant body of knowledge about the antecedents and consequences of individual differences in belief in conspiracy theories. What is less clear, however, is the extent to which individuals’ beliefs in con

Externí odkaz: https://doaj.org/article/a2fdc925fe4f4646b00c804d73b76db5

Impact of COVID-19 on heart failure hospitalization and outcome in India – A cardiological society of India study (CSI–HF in COVID 19 times study – 'The COVID C–HF study')

Autor: Jayagopal P. B, Ramakrishnan S, Mohanan P. P, Jabir A, Venugopal K, M.K. Das, Santhosh K, Syam N, J. Ezhilan, Rajeev Agarwal, Vaidyanathan P R, Ahmed Hussain Choudhary, Meena C B, Amit Malviya, Arun Gopi, Chopra V K, Stigi Joseph, Krishna Kishore Goyal, John F. John, Sandeep Bansal, Harikrishnan S, Praveen Nagula, Johny Joseph, Ajit Bagawat, Sandeep Seth, Urmil Shah, Pravin K. Goel, P.K. Asokan, K.K. Sethi, Satyavan Sharma, (Lt.Gen)Anup Banerji, Sunandan Sikdar, ManojKumar Agarwala, Sharad Chandra, BishwaBhushan Bharti, S.M. Ashraf, Smit Srivastava, B. Kesavamoorthy, Harinder Kumar Bali, Dipak Sarma, Rajendra Kumar Jain, Sameer I. Dani, B.H. Natesh, Rabindra Nath Chakraborty, Vivek Gupta, Narendra Nath Khanna, Dipankar Mukhopadhyay, Subroto Mandal, Biswajit Majumder, Sridhar L, Meennahalli Palleda Girish, DipakRanjan Das, Tom Devasia, Bhavesh Vajifdar, Tanuj Bhatia, Zia Abdullah, Sanjeev Sharma, Sudeep Kumar, Mathew Lincy, Nitish Naik, Dhiman Kahali, Dhurjati Prasad Sinha, Dipankar Ghosh Dastidar, Gurpreet Sing Wander, Rakesh Yadav, Satyendra Tewari, Suman Bhandari, Pratap Chandra Rath, Vijay Harikisan Bang, Debabrata Roy, ParthoSarathi Banerjee, S. Shanmugasundaram, Geevar Zachariah

Publikováno v: Indian Heart Journal, Vol 75, Iss 5, Pp 370-375 (2023)

Objectives: The presentation and outcomes of acute decompensated heart failure (ADHF) during COVID times (June 2020 to Dec 2020) were compared with the historical control during the same period in 2019. Methods: Data of 4806 consecutive patients of a

Externí odkaz: https://doaj.org/article/3b8a535b96fb4971af395995c76eeeb4

Different neural crest populations exhibit diverse proliferative behaviors

Autor: Lincon A. Stamp, Heather M. Young, Mathew Li-Yuen-Fong, David G. Gonsalvez, Kylie N. Cane, Colin R. Anderson

Publikováno v: Developmental Neurobiology. 75:287-301

The rate of proliferation of cells depends on the proportion of cycling cells and the frequency of cell division. Here, we describe in detail methods for quantifying the proliferative behavior of specific cell types in situ, and use the method to exa

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::ce17154d187f5ead580d8f45bde33014
https://doi.org/10.1002/dneu.22229