Agonist-mediated switching of ion selectivity in TPC2 differentially promotes lysosomal function.
Elife
Gerndt, Susanne S; Chen, Cheng-Chang CC; Chao, Yu-Kai YK; Yuan, Yu Y; Burgstaller, Sandra S; Scotto Rosato, Anna A; Krogsaeter, Einar E; Urban, Nicole N; Jacob, Katharina K; Nguyen, Ong Nam Phuong ONP; Miller, Meghan T MT; Keller, Marco M; Vollmar, Angelika M AM; Gudermann, Thomas T; Zierler, Susanna S; Schredelseker, Johann J; Schaefer, Michael M; Biel, Martin M; Malli, Roland R; Wahl-Schott, Christian C; Bracher, Franz F; Patel, Sandip S; Grimm, Christian C
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia.
Nature Communications
Mettananda, Sachith S; Fisher, Chris A CA; Hay, Deborah D; Badat, Mohsin M; Quek, Lynn L; Clark, Kevin K; Hublitz, Philip P; Downes, Damien D; Kerry, Jon J; Gosden, Matthew M; Telenius, Jelena J; Sloane-Stanley, Jackie A JA; Faustino, Paula P; Coelho, Andreia A; Doondeea, Jessica J; Usukhbayar, Batchimeg B; Sopp, Paul P; Sharpe, Jacqueline A JA; Hughes, Jim R JR; Vyas, Paresh P; Gibbons, Richard J RJ; Higgs, Douglas R DR
The nucleoside analog clitocine is a potent and efficacious readthrough agent.
Rna (New York, N.Y.)
Friesen, Westley J WJ; Trotta, Christopher R CR; Tomizawa, Yuki Y; Zhuo, Jin J; Johnson, Briana B; Sierra, Jairo J; Roy, Bijoyita B; Weetall, Marla M; Hedrick, Jean J; Sheedy, Josephine J; Takasugi, James J; Moon, Young-Choon YC; Babu, Suresh S; Baiazitov, Ramil R; Leszyk, John D JD; Davis, Thomas W TW; Colacino, Joseph M JM; Peltz, Stuart W SW; Welch, Ellen M EM