未登录 Design of Heart-2: a phase 1b clinical trial of VERVE-102, an in vivo base editing medicine delivered by a GalNAc-LNP and targeting PCSK9 to durably lower LDL cholesterol Heart-2的设计：一项关于VERVE-102的1b期临床试验，VERVE-102是一种通过GalNAc-LNP递送的体内碱基编辑药物，靶向PCSK9以持久降低LDL胆固醇 Vafai, Scott; Karsten, Verena; Jensen, Chelsey; Falzone, Richard; Lister, Troy; Stolz, Leslie; Khera, Amit; Kathiresan, Sekar; Bellinger, Andrew; Fiedorek, Fred 分享 收藏
Spacer Fidelity Assessments of Guide RNA by Top-Down Mass Spectrometry Macias, Luis A. A.; Garcia, Sara P. P.; Back, Kayla M. M.; Wu, Yue; Johnson, G. Hall; Kathiresan, Sekar; Bellinger, Andrew M. M.; Rohde, Ellen; Freitas, Michael A. A.; Madsen, James A. A. 分享 收藏
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GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy Kasiewicz, Lisa N.; Biswas, Souvik; Beach, Aaron; Ren, Huilan; Dutta, Chaitali; Mazzola, Anne Marie; Rohde, Ellen; Chadwick, Alexandra; Cheng, Christopher; Garcia, Sara P.; Iyer, Sowmya; Matsumoto, Yuri; Khera, Amit V.; Musunuru, Kiran; Kathiresan, Sekar; Malyala, Padma; Rajeev, Kallanthottathil G.; Bellinger, Andrew M. 分享 收藏
Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models 针对PCSK9的研究性单程CRISPR碱基编辑疗法在非人灵长类动物和小鼠模型中的疗效和安全性 Lee, Richard G.; Mazzola, Anne Marie; Braun, Maurine C.; Platt, Colin; Vafai, Scott B.; Kathiresan, Sekar; Rohde, Ellen; Bellinger, Andrew M.; Khera, Amit, V 分享 收藏
Discovery and systematic characterization of risk variants and genes for coronary artery disease in over a million participants 超过一百万参与者的冠状动脉疾病风险变异和基因的发现和系统表征 Aragam, Krishna G.; Jiang, Tao; Goel, Anuj; Kanoni, Stavroula; Wolford, Brooke N.; Atri, Deepak S.; Weeks, Elle M.; Wang, Minxian; Hindy, George; Zhou, Wei; Grace, Christopher; Roselli, Carolina; Marston, Nicholas A.; Kamanu, Frederick K.; Surakka, Ida; Venegas, Loreto Munoz; Sherliker, Paul; Koyama, Satoshi; Ishigaki, Kazuyoshi; Asvold, Bjorn O.; Brown, Michael R.; Brumpton, Ben; de Vries, Paul S.; Giannakopoulou, Olga; Giardoglou, Panagiota; Gudbjartsson, Daniel F.; Gueldener, Ulrich; Haider, Syed M. Ijlal; Helgadottir, Anna; Ibrahim, Maysson; Kastrati, Adnan; Kessler, Thorsten; Kyriakou, Theodosios; Konopka, Tomasz; Li, Ling; Ma, Lijiang; Meitinger, Thomas; Mucha, Soeren; Munz, Matthias; Murgia, Federico; Nielsen, Jonas B.; Noethen, Markus M.; Pang, Shichao; Reinberger, Tobias; Schnitzler, Gavin; Smedley, Damian; Thorleifsson, Gudmar; von Scheidt, Moritz; Ulirsch, Jacob C.; Arnar, David O.; Burtt, Noel P.; Costanzo, Maria C.; Flannick, Jason; Ito, Kaoru; Jang, Dong-Keun; Kamatani, Yoichiro; Khera, Amit V.; Komuro, Issei; Kullo, Iftikhar J.; Lotta, Luca A.; Nelson, Christopher P.; Roberts, Robert; Thorgeirsson, Gudmundur; Thorsteinsdottir, Unnur; Webb, Thomas R.; Baras, Aris; Bjoerkegren, Johan L. M.; Boerwinkle, Eric; Dedoussis, George; Holm, Hilma; Hveem, Kristian; Melander, Olle; Morrison, Alanna C.; Orho-Melander, Marju; Rallidis, Loukianos S.; Ruusalepp, Arno; Sabatine, Marc S.; Stefansson, Kari; Zalloua, Pierre; Ellinor, Patrick T.; Farrall, Martin; Danesh, John; Ruff, Christian T.; Finucane, Hilary K.; Hopewell, Jemma C.; Clarke, Robert; Gupta, Rajat M.; Erdmann, Jeanette; Samani, Nilesh J.; Schunkert, Heribert; Watkins, Hugh; Willer, Cristen J.; Deloukas, Panos; Kathiresan, Sekar; Butterworth, Adam S. 分享 收藏
Gene Sequencing Identifies Perturbation in Nitric Oxide Signaling as a Nonlipid Molecular Subtype of Coronary Artery Disease Khera, Amit, V; Wang, Minxian; Chaffin, Mark; Emdin, Connor A.; Samani, Nilesh J.; Schunkert, Heribert; Watkins, Hugh; McPherson, Ruth; Erdmann, Jeanette; Elosua, Roberto; Boerwinkle, Eric; Ardissino, Diego; Butterworth, Adam S.; Di Angelantonio, Emanuele; Naheed, Aliya; Danesh, John; Chowdhury, Rajiv; Krumholz, Harlan M.; Sheu, Wayne H-H; Rich, Stephen S.; Rotter, Jerome, I; Chen, Yii-der Ida; Gabriel, Stacey; Lander, Eric S.; Saleheen, Danish; Kathiresan, Sekar 分享 收藏
Neurocognitive trajectory and proteomic signature of inherited risk for Alzheimer's disease Paranjpe, Manish D.; Chaffin, Mark; Zahid, Sohail; Ritchie, Scott; Rotter, Jerome, I; Rich, Stephen S.; Gerszten, Robert; Guo, Xiuqing; Heckbert, Susan; Tracy, Russ; Danesh, John; Lander, Eric S.; Inouye, Michael; Kathiresan, Sekar; Butterworth, Adam S.; Khera, Amit, V 分享 收藏
Rare coding variants in 35 genes associate with circulating lipid levels-A multi-ancestry analysis of 170,000 exomes 35个基因的罕见编码变异与循环脂质水平相关 -- 170,000外显子组的多祖先分析 Hindy, George; Dornbos, Peter; Chaffin, Mark D.; Liu, Dajiang J.; Wang, Minxian; Selvaraj, Margaret Sunitha; Zhang, David; Park, Joseph; Aguilar-Salinas, Carlos A.; Antonacci-Fulton, Lucinda; Ardissino, Diego; Arnett, Donna K.; Aslibekyan, Stella; Atzmon, Gil; Ballantyne, Christie M.; Barajas-Olmos, Francisco; Barzilai, Nir; Becker, Lewis C.; Bielak, Lawrence F.; Bis, Joshua C.; Blangero, John; Boerwinkle, Eric; Bonnycastle, Lori L.; Bottinger, Erwin; Bowden, Donald W.; Bown, Matthew J.; Brody, Jennifer A.; Broome, Jai G.; Burtt, Noel P.; Cade, Brian E.; Centeno-Cruz, Federico; Chan, Edmund; Chang, Yi-Cheng; Chen, Yii-Der I.; Cheng, Ching-Yu; Choi, Won Jung; Chowdhury, Rajiv; Contreras-Cubas, Cecilia; Cordova, Emilio J.; Correa, Adolfo; Cupples, L. Adrienne; Curran, Joanne E.; Danesh, John; de Vries, Paul S.; DeFronzo, Ralph A.; Doddapaneni, Harsha; Duggirala, Ravindranath; Dutcher, Susan K.; Ellinor, Patrick T.; Emery, Leslie S.; Florez, Jose C.; Fornage, Myriam; Freedman, Barry I.; Fuster, Valentin; Garay-Sevilla, Ma. Eugenia; Garcia-Ortiz, Humberto; Germer, Soren; Gibbs, Richard A.; Gieger, Christian; Glaser, Benjamin; Gonzalez, Clicerio; Gonzalez-Villalpando, Maria Elena; Graff, Mariaelisa; Graham, Sarah E.; Grarup, Niels; Groop, Leif C.; Guo, Xiuqing; Gupta, Namrata; Han, Sohee; Hanis, Craig L.; Hansen, Torben; He, Jiang; Heard-Costa, Nancy L.; Hung, Yi-Jen; Hwang, Mi Yeong; Irvin, Marguerite R.; Islas-Andrade, Sergio; Jarvik, Gail P.; Kang, Hyun Min; Kardia, Sharon L. R.; Kelly, Tanika; Kenny, Eimear E.; Khan, Alyna T.; Kim, Bong-Jo; Kim, Ryan W.; Kim, Young Jin; Koistinen, Heikki A.; Kooperberg, Charles; Kuusisto, Johanna; Kwak, Soo Heon; Laakso, Markku; Lange, Leslie A.; Lee, Jiwon; Lee, Juyoung; Lee, Seonwook; Lehman, Donna M.; Lemaitre, Rozenn N.; Linneberg, Allan; Liu, Jianjun; Loos, Ruth J. F.; Lubitz, Steven A.; Lyssenko, Valeriya; Ma, Ronald C. W.; Martin, Lisa Warsinger; Martinez-Hernandez, Angelica; Mathias, Rasika A.; McGarvey, Stephen T.; McPherson, Ruth; Meigs, James B.; Meitinger, Thomas; Melander, Olle; Mendoza-Caamal, Elvia; Metcalf, Ginger A.; Mi, Xuenan; Mohlke, Karen L.; Montasser, May E.; Moon, Jee-Young; Moreno-Macias, Hortensia; Morrison, Alanna C.; Muzny, Donna M.; Nelson, Sarah C.; Nilsson, Peter M.; O'Connell, Jeffrey R.; Orho-Melander, Marju; Orozco, Lorena; Palmer, Colin N. A.; Palmer, Nicholette D.; Park, Cheol Joo; Park, Kyong Soo; Pedersen, Oluf; Peralta, Juan M.; Peyser, Patricia A.; Post, Wendy S.; Preuss, Michael; Psatsy, Bruce M.; Qi, Qibin; Rao, D. C.; Redline, Susan; Reiner, Alexander P.; Revilla-Monsalve, Cristina; Rich, Stephen S.; Samani, Nilesh; Schunkert, Heribert; Schurmann, Claudia; Seo, Daekwan; Seo, Jeong-Sun; Sim, Xueling; Sladek, Rob; Small, Kerrin S.; So, Wing Yee; Stilp, Adrienne M.; Tai, E. Shyong; Tam, Claudia H. T.; Taylor, Kent D.; Teo, Yik Ying; Thameem, Farook; Tomlinson, Brian; Tsai, Michael Y.; Tuomi, Tiinamaij; Tuomilehto, Jaakko; Tusie-Luna, Teresa; Udler, Miriam S.; van Dam, Rob M.; Vasan, Ramachandran S.; Martinez, Karine A. Viaud; Wang, Fei Fei; Wang, Xuzhi; Watkins, Hugh; Weeks, Daniel E.; Wilson, James G.; Witte, Daniel R.; Wong, Tien-Yin; Yanek, Lisa R.; Kathiresan, Sekar; Rader, Daniel J.; Rotter, Jerome I.; Boehnke, Michael; McCarthy, Mark I.; Willer, Cristen J.; Natarajan, Pradeep; Flannick, Jason A.; Khera, Amit V.; Peloso, Gina M. 分享 收藏
Integrative analysis of the plasma proteome and polygenic risk of cardiometabolic diseases Ritchie, Scott C.; Lambert, Samuel A.; Arnold, Matthew; Teo, Shu Mei; Lim, Sol; Scepanovic, Petar; Marten, Jonathan; Zahid, Sohail; Chaffin, Mark; Liu, Yingying; Abraham, Gad; Ouwehand, Willem H.; Roberts, David J.; Watkins, Nicholas A.; Drew, Brian G.; Calkin, Anna C.; Di Angelantonio, Emanuele; Soranzo, Nicole; Burgess, Stephen; Chapman, Michael; Kathiresan, Sekar; Khera, Amit, V; Danesh, John; Butterworth, Adam S.; Inouye, Michael 分享 收藏
Rare, Damaging DNA Variants in CORIN and Risk of Coronary Artery Disease Insights From Functional Genomics and Large-Scale Sequencing Analyses Wang, Minxian; Lee-Kim, Vivian S.; Atri, Deepak S.; Elowe, Nadine H.; Yu, John; Garvie, Colin W.; Won, Hong-Hee; Hadaya, Joseph E.; MacDonald, Bryan T.; Trindade, Kevin; Melander, Olle; Rader, Daniel J.; Natarajan, Pradeep; Kathiresan, Sekar; Kaushik, Virendar K.; Khera, Amit V.; Gupta, Rajat M. 分享 收藏
Coronary Disease Association With ADAMTS7 Is Due to Protease Activity Mizoguchi, Taiji; MacDonald, Bryan T.; Bhandary, Bidur; Popp, Nicholas R.; Laprise, Dylan; Arduini, Alessandro; Lai, Daniel; Zhu, Qiuyu Martin; Xing, Yi; Kaushik, Virendar K.; Kathiresan, Sekar; Ellinor, Patrick T. 分享 收藏
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A missense variant in Mitochondrial Amidoxime Reducing Component 1 gene and protection against liver disease (vol 16, e1008629, 2020) 线粒体偕胺肟还原成分1基因的错义变体和对肝脏疾病的保护 (第16卷，e1008629，2020) Emdin, Connor A.; Haas, Mary E.; Khera, Amit V.; Aragam, Krishna; Chaffin, Mark; Klarin, Derek; Hindy, George; Jiang, Lan; Wei, Wei-Qi; Feng, Qiping; Karjalainen, Juha; Havulinna, Aki; Kiiskinen, Tuomo; Bick, Alexander; Ardissino, Diego; Wilson, James G.; Schunkert, Heribert; McPherson, Ruth; Watkins, Hugh; Elosua, Roberto; Bown, Matthew J.; Samani, Nilesh J.; Baber, Usman; Erdmann, Jeanette; Gupta, Namrata; Danesh, John; Saleheen, Danish; Chang, Kyong-Mi; Vujkovic, Marijana; Voight, Ben; Damrauer, Scott; Lynch, Julie; Kaplan, David; Serper, Marina; Tsao, Philip; Program, Million Veteran; Mercader, Josep; Hanis, Craig; Daly, Mark; Denny, Joshua; Gabriel, Stacey; Kathiresan, Sekar 分享 收藏
Association of Genetic Variation With Cirrhosis: A Multi-Trait Genome-Wide Association and Gene-Environment Interaction Study Emdin, Connor A.; Haas, Mary; Ajmera, Veeral; Simon, Tracey G.; Homburger, Julian; Neben, Cynthia; Jiang, Lan; Wei, Wei-Qi; Feng, Qiping; Zhou, Alicia; Denny, Joshua; Corey, Kathleen; Loomba, Rohit; Kathiresan, Sekar; Khera, Amit, V 分享 收藏
Genome-Wide Polygenic Score, Clinical Risk Factors, and Long-Term Trajectories of Coronary Artery Disease Hindy, George; Aragam, Krishna G.; Ng, Kenney; Chaffin, Mark; Lotta, Luca A.; Baras, Aris; Drake, Isabel; Orho-Melander, Marju; Melander, Olle; Kathiresan, Sekar; Khera, Amit V. 分享 收藏
Genetic Architecture of Abdominal Aortic Aneurysm in the Million Veteran Program Klarin, Derek; Verma, Shefali Setia; Judy, Renae; Dikilitas, Ozan; Wolford, Brooke N.; Paranjpe, Ishan; Levin, Michael G.; Pan, Cuiping; Tcheandjieu, Catherine; Spin, Joshua M.; Lynch, Julie; Assimes, Themistocles L.; Aldstedt Nyronning, Linn; Mattsson, Erney; Edwards, Todd L.; Denny, Josh; Larson, Eric; Lee, Ming Ta Michael; Carrell, David; Zhang, Yanfei; Jarvik, Gail P.; Gharavi, Ali G.; Harley, John; Mentch, Frank; Pacheco, Jennifer A.; Hakonarson, Hakon; Skogholt, Anne Heidi; Thomas, Laurent; Gabrielsen, Maiken Elvestad; Hveem, Kristian; Nielsen, Jonas Bille; Zhou, Wei; Fritsche, Lars; Huang, Jie; Natarajan, Pradeep; Sun, Yan V.; DuVall, Scott L.; Rader, Daniel J.; Cho, Kelly; Chang, Kyong-Mi; Wilson, Peter W. F.; O'Donnell, Christopher J.; Kathiresan, Sekar; Scali, Salvatore T.; Berceli, Scott A.; Willer, Cristen; Jones, Gregory T.; Bown, Matthew J.; Nadkarni, Girish; Kullo, Iftikhar J.; Ritchie, Marylyn; Damrauer, Scott M.; Tsao, Philip S. 分享 收藏
Heterozygous ABCG5 Gene Deficiency and Risk of Coronary Artery Disease Nomura, Akihiro; Emdin, Connor A.; Won, Hong Hee; Peloso, Gina M.; Natarajan, Pradeep; Ardissino, Diego; Danesh, John; Schunkert, Heribert; Correa, Adolfo; Bown, Matthew J.; Samani, Nilesh J.; Erdmann, Jeanette; McPherson, Ruth; Watkins, Hugh; Saleheen, Danish; Elosua, Roberto; Kawashiri, Masa-aki; Tada, Hayato; Gupta, Namrata; Shah, Svati H.; Rader, Daniel J.; Gabriel, Stacey; Khera, Amit V.; Kathiresan, Sekar 分享 收藏
Rare variants lowering the levels of coagulation factor X are protective against ischemic heart disease Paraboschi, Elvezia Maria; Khera, Amit Vikram; Merlini, Piera Angelica; Gigante, Laura; Peyvandi, Flora; Chaffin, Mark; Menegatti, Marzia; Busti, Fabiana; Girelli, Domenico; Martinelli, Nicola; Olivieri, Oliviero; Kathiresan, Sekar; Ardissino, Diego; Asselta, Rosanna; Duga, Stefano 分享 收藏
ALK-7: A Validated Target for the Treatment of Obesity Bu, Yun; Zhao, Min; Fredericks, Maureen; Cannell, Marishka; Dagon, Yossi; Emdin, Connor A.; Okunishi, Katsuhide; Wang, Hao; Sako, Dianne; Castonguay, Roselyne; Suragani, Rajasekhar N. V.; Kathiresan, Sekar; Grinberg, Asya; Knopf, John; Pearsall, Robert S.; Kumar, Ravindra; Izumi, Tetsuro 分享 收藏
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