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作者:Bulcaen, Mattijs; Carlon, Marianne S.
作者单位:KU Leuven
摘要:The lungs encompass a vast surface for gas exchange-they are the bellows that keep the body oxygenated and its metabolism burning. When problems arise in the lungs, severe diseases follow, such as cystic fibrosis. Development of gene therapies for cystic fibrosis has been challenging, especially because delivery usually involves inhalation. Permanently exposed to the hostile outside environment, the lungs are equipped with defense mechanisms to keep inhaled particles and pathogens out of the l...
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作者:Sun, Yehui; Chatterjee, Sumanta; Lian, Xizhen; Traylor, Zachary; Sattiraju, Sandhya R.; Xiao, Yufen; Dilliard, Sean A.; Sung, Yun-Chieh; Kim, Minjeong; Lee, Sang M.; Moore, Stephen; Wang, Xu; Zhang, Di; Wu, Shiying; Basak, Pratima; Wang, Jialu; Liu, Jing; Mann, Rachel J.; Lepage, David F.; Jiang, Weihong; Abid, Shadaan; Hennig, Mirko; Martinez, Anna; Wustman, Brandon A.; Lockhart, David J.; Jain, Raksha; Conlon, Ronald A.; Drumm, Mitchell L.; Hodges, Craig A.; Siegwart, Daniel J.
作者单位:University of Texas System; University of Texas Southwestern Medical Center; University System of Ohio; Case Western Reserve University; University of Texas System; University of Texas Southwestern Medical Center
摘要:In vivo genome correction holds promise for generating durable disease cures; yet, effective stem cell editing remains challenging. In this work, we demonstrate that optimized lung-targeting lipid nanoparticles (LNPs) enable high levels of genome editing in stem cells, yielding durable responses. Intravenously administered gene-editing LNPs in activatable tdTomato mice achieved >70% lung stem cell editing, sustaining tdTomato expression in >80% of lung epithelial cells for 660 days. Addressing...
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作者:Couzin-Frankel, Jennifer
摘要:Pivotal trial of Eli Lilly's donanemab leaves questions about how to prescribe it and minimize risks
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作者:Mohammadi, Alireza; Azareh, Ali; Rafiei-Sardooi, Elham; Khanjani, Mohammad Hossein
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作者:Huang, Qin; Chan, Ken Y.; Wu, Jason; Botticello-Romero, Nuria R.; Zheng, Qingxia; Lou, Shan; Keyes, Casey; Svanbergsson, Alexander; Johnston, Jencilin; Mills, Allan; Lin, Chin-Yen; Brauer, Pamela P.; Clouse, Gabrielle; Pacouret, Simon; Harvey, John W.; Beddow, Thomas; Hurley, Jenna K.; Tobey, Isabelle G.; Powell, Megan; Chen, Albert T.; Barry, Andrew J.; Eid, Fatma-Elzahraa; Chan, Yujia A.; Deverman, Benjamin E.
作者单位:Harvard University; Massachusetts Institute of Technology (MIT); Broad Institute; Egyptian Knowledge Bank (EKB); Al Azhar University
摘要:Developing vehicles that efficiently deliver genes throughout the human central nervous system (CNS) will broaden the range of treatable genetic diseases. We engineered an adeno-associated virus (AAV) capsid, BI-hTFR1, that binds human transferrin receptor (TfR1), a protein expressed on the blood-brain barrier. BI-hTFR1 was actively transported across human brain endothelial cells and, relative to AAV9, provided 40 to 50 times greater reporter expression in the CNS of human TFRC knockin mice. ...
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作者:Machlis, Gary E.; Carrero-Martinez, Franklin A.
作者单位:Clemson University; National Academies of Sciences, Engineering & Medicine
摘要:Since 2001, the United Nations has designated 20 June as World Refugee Day. It seeks to raise awareness of refugees-currently estimated to be more than 114 million persons internally displaced, exiled, or on the move-and the often-harsh conditions they confront around the world. Among them are scientists, giving the scientific community a special responsibility to protect and support these colleagues. It is not just scientists and their families whose lives are disrupted, but science as an ent...
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作者:Selibas, Dimitri
摘要:CyberTracker, software developed in collaboration with Indigenous trackers, is enabling almost anyone to collect complex biodiversity data
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作者:Warren, Matthew
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作者:Duan, J.; Cloete, R.; Loock, J. C.; Lanzirotti, A.; Newville, M.; Martinez-Garcia, A.; Sigman, D. M.; Lam, P. J.; Roychoudhury, A. N.; Myneni, S. C. B.
作者单位:Princeton University; Stellenbosch University; University of Chicago; Max Planck Society; University of California System; University of California Santa Cruz
摘要:Zinc (Zn) is vital to marine organisms. Its active uptake by phytoplankton results in a substantial depletion of dissolved Zn, and Zn bound to particulate organic matter replenishes dissolved Zn in the ocean through remineralization. However, we found that particulate Zn changes from Zn bound to phosphoryls in cells to recalcitrant inorganic pools that include biogenic silica, clays, and iron, manganese, and aluminum oxides in the Southern Ocean water column. The abundances of inorganic pools ...
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作者:Halpern, Paul
作者单位:Saint Joseph's University
摘要:A physicist makes the case for a deterministic alternative to quantum mechanics