Pharmacokinetics of hydroxychloroquine in paediatric lupus: data from a novel, direct-to-family clinical trial

Autor: E Schmitt, C Smith, G Schulert, S Canna, A Grom, E Mellins, A Brown, J Smith, A Stevens, M Watson, S Jones, K Stewart, E Baker, A Kemp, T Davis, A Smith, S Jackson, C Williams, K Jones, T Mason, A Hanson, Y Zhao, B Thomas, A Reed, J Jones, J Cooper, T Lee, J Chang, M Holland, S Joshi, L Lim, C Ramírez, A Murphy, K Moore, E Pagano, B ferreira, S Li, P Lee, H Schmeling, K Abulaban, R Agbayani, S Akoghlanian, E Anderson, L Barillas-Arias, K Baszis, M Becker, H Bell-Brunson, H Benham, S Benseler, T Beukelman, H Brunner, H Bukulmez, L Cerracchio, E Chalom, K Chundru, J Dean, F Dedeoglu, V Dempsey, J Drew, B Feldman, P Ferguson, C Fleming, L Franco, I Goh, D Goldsmith, B Gottlieb, T Graham, T Griffin, M Hance, K Hickey, M Hollander, J Hsu, A Huber, C Hung, A Huttenlocher, L Imundo, C Inman, J Jaquith, L Jung, D Kingsbury, K Klein, M Klein-Gitelman, S Kramer, S Lapidus, D Latham, B Malla, M Malloy, A Martyniuk, K McConnell, D McCurdy, C McMullen-Jackson, L Moorthy, E Muscal, J Olson, K Onel, L Ponder, S Prahalad, C Rabinovich, S Ringold, M Riordan, A Robinson, M Rosenkranz, B Rosolowski, N Ruth, K Schikler, H Stapp, R Syed, M Tesher, A Thatayatikom, R Vehe, E von Scheven, D Wahezi, A Watts, J Weiss, J Wagner, S Kim, Y Zhang, L Favier, J Patel, S Morgan, A Jackson, J Stokes, L Marques, Stephen J Balevic, K Hayward, A White, J Nicholas, D Lovell, A Zeft, J Harris, E Lawson, C Moss, N George, M Sutter, A Cooper, M Adams, S Cooper, M Miller, C Black, R Schneider, J Taylor, R Sran, M Oliver, M Twilt, M Tóth, J Walker, M Mitchell, F De Benedetti, N Singer, M Fox, K Kaufman, A Merritt, R Stevenson, J Fuller, M Fitzgerald, A Davis, C Davis, L Henderson, J Woo, S Mohan, H Reid, Y Kimura, L Harel, R Laxer, K McCarthy, I Ferguson, E McCormick, A Hay, M Guzman, E Fox, P Hill, A PARSONS, S McGuire, J Lam, C Sandborg, B Stevens, J Boland, S Ballinger, E MENDOZA, J NOCTON, M Ritter, N Johnson, J Shirley, S Bowman, M Ibarra, S Hong, M Guevara, K James, L Santiago, A Adams, B DONALDSON, M Son, C Kremer, K Schmidt, T Wright, L Cannon, R Nicolai, M Freeman, S Spence, D Levy, J Paredes, K Gerhold, A Insalaco, T O'Brien, W Bernal, E Kessler, C Lin, M Lerman, T Hahn, B O'Brien, Michael Cohen-Wolkowiez, Christoph P Hornik, N Abel, J Aiello, C Alejandro, E Allenspach, R Alperin, M Alpizar, G Amarilyo, W Ambler, S Ardoin, S Armendariz, I Balboni, S Balevic, L Ballenger, N Balmuri, F Barbar-Smiley, M Basiaga, E Beltz, T Bigley, B Binstadt, M Blakley, J Bohnsack, A Boneparth, C Bracaglia, E Brooks, M Brothers, M Buckley, D Bullock, B Cameron, P Carper, V Cartwright, E Cassidy, A Chang-Hoftman, V Chauhan, P Chira, T Chinn, H Clairman, D Co, A Confair, H Conlon, R Connor, C Correll, R Corvalan, D Costanzo, R Cron, L Curiel-Duran, T Curington, M Curry, A Dalrymple, D De Ranieri, M De Guzman, N Delnay, E DeSantis, T Dickson, J Dingle, E Dorsey, S Dover, J Dowling, K Driest, Q Du, K Duarte, D Durkee, E Duverger, J Dvergsten, A Eberhard, M Eckert, K Ede, B Edelheit, C Edens, Y Edgerly, M Elder, B Ervin, S Fadrhonc, C Failing, D Fair, M Falcon, S Federici, J Fennell, R Ferrucho, K Fields, T Finkel, O Flynn, L Fogel, K Fritz, S Froese, R Fuhlbrigge, D Gerstbacher, M Gilbert, M Gillispie-Taylor, E Giverc, C Godiwala, H Goheer, E Gotschlich, A Gotte, C Gracia, S Grevich, J Griswold, P Guittar, M Hager, O Halyabar, E Hammelev, S Haro, O Harry, E Hartigan, J Hausmann, J Heiart, K Hekl, M Henrickson, A Hersh, S Hillyer, L Hiraki, M Hiskey, P Hobday, C Hoffart, M Horwitz, J Huggins, J HuiYuen, J Huntington, G Janow, S Jared, C Justice, A Justiniano, N Karan, U Khalsa, B Kienzle, M Kitcharoensakkul, T Klausmeier, B Kompelien, A Kosikowski, L Kovalick, J Kracker, J Lai, B Lang, B Lapin, A Lasky, L Lentini, S Lieberman, N Ling, M Lingis, M Lo, D Lowman, N Luca, S Lvovich, C Madison, J Madison, S Magni Manzoni, J Maller, M Mannion, C Manos, S Mathus, L McAllister, P McCurdy Stokes, I McHale, A McMonagle, E Meidan, R Mercado, L Michalowski, P Miettunen, D Milojevic, E Mirizio, E Misajon, R Modica, E Morgan Dewitt, T Moussa, V Mruk, R Nadler, B Nahal, K Nanda, N Nasah, L Nassi, S Nativ, M Natter, J Neely, B Nelson, L Newhall, L Ng, P Nigrovic, B Nolan, E Oberle, B Obispo, O Okeke, K O'Neil, A Orandi, M Orlando, S Osei-Onomah, R Oz, A Paller, N Pan, S Panupattanapong, M Pardeo, K Pentakota, P Pepmueller, T Pfeiffer, K Phillippi, D Pires Marafon, R Pooni, S Pratt, S Protopapas, B Puplava, J Quach, M Quinlan-Waters, S Radhakrishna, J Rafko, J Raisian, A Rakestraw, E Ramsay, S Ramsey, R Randell, K Remmel, A Repp, A Reyes, A Richmond, M Riebschleger, M Riskalla, R Rivas-Chacon, E Rodela, M Rodriquez, K Rojas, T Ronis, H Rothermel, D Rothman, E Roth-Wojcicki, K Rouster-Stevens, T Rubinstein, N Saad, S Sabbagh, E Sacco, R Sadun, A Sanni, A Sarkissian, S Savani, L Scalzi, L Schanberg, S Scharnhorst, A Schlefman, K Schollaert-Fitch, T Seay, C Seper, J Shalen, R Sheets, A Shelly, S Shenoi, K Shergill, M Shishov, C Shivers, E Silverman, V Sivaraman, J Sletten, E Smitherman, J Soep, L Spiegel, J Spitznagle, H Srinivasalu, K Steigerwald, Y Sterba Rakovchik, S Stern, C Stingl, M Stoll, E Stringer, S Sule, J Sumner, R Sundel, G Syverson, A Szymanski, S Taber, R Tal, A Tambralli, A Taneja, T Tanner, S Tapani, G Tarshish, S Tarvin, L Tate, A Taxter, M Terry, K Tiffany, T Ting, A Tipp, D Toib, K Torok, C Toruner, H Tory, S Tse, V Tubwell, S Uriguen, T Valcarcel, H Van Mater, L Vannoy, C Varghese, N Vasquez, K Vazzana, K Veiga, J Velez, J Verbsky, G Vilar, N Volpe, S Vora, L Wagner-Weiner, H Waite, H Walters, T Wampler Muskardin, L Waqar, M Waterfield, P Weiser, P Weiss, E Wershba, A Wise, L Woolnough, E Wu, A Yalcindag, M Yee, E Yen, R Yeung, K Yomogida, Q Yu, R Zapata, A Zartoshti, R Zeft, A Zhu, C Zic, Daniel Weiner, Daniel Gonzalez, Rachel Randell, Claire Beard

Publikováno v: Lupus Science and Medicine, Vol 9, Iss 1 (2022)

Objective Determine the pharmacokinetics (PK) and exposure–response of hydroxychloroquine (HCQ) and desethylhydroxychloroquine (DHCQ) in paediatric SLE (pSLE).Methods We conducted an exploratory phase 2, direct-to-family trial. Children enrolled in

Externí odkaz: https://doaj.org/article/20ce5e9cb2a841b29d1ddbfe3a1ed845

‘Cold shock’ increases the frequency of homology directed repair gene editing in induced pluripotent stem cells

Autor: Q. Guo, G. Mintier, M. Ma-Edmonds, D. Storton, X. Wang, X. Xiao, B Kienzle, D. Zhao, John N. Feder

Publikováno v: Scientific Reports, Vol 8, Iss 1, Pp 1-11 (2018)

Abstract Using CRISPR/Cas9 delivered as a RNA modality in conjunction with a lipid specifically formulated for large RNA molecules, we demonstrate that homology directed repair (HDR) rates between 20–40% can be achieved in induced pluripotent stem

Externí odkaz: https://doaj.org/article/7f2a87b2a216424889cfd9d640ef048d

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‘Cold shock’ increases the frequency of homology directed repair gene editing in induced pluripotent stem cells

Autor: D Storton, Gabe Mintier, Qi Guo, M Ma-Edmonds, Xuning Wang, D Zhao, John N. Feder, B Kienzle, X Xiao

Publikováno v: Scientific Reports
Scientific Reports, Vol 8, Iss 1, Pp 1-11 (2018)

Using CRISPR/Cas9 delivered as a RNA modality in conjunction with a lipid specifically formulated for large RNA molecules, we demonstrate that homology directed repair (HDR) rates between 20–40% can be achieved in induced pluripotent stem cells (iP

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::26bdb454b8e498b1725539f3634d9b00
https://doi.org/10.1038/s41598-018-20358-5

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