Gene therapy targeting protein trafficking regulator MOG1 in mouse models of Brugada syndrome, arrhythmias, and mild cardiomyopathy

JOURNAL ARTICLE published 8 June 2022 in Science Translational Medicine

Authors: Gang Yu | Susmita Chakrabarti | Miroslava Tischenko | Ai-Lan Chen | Zhijie Wang | Hyosuk Cho | Brent A. French | Sathyamangla V. Naga Prasad | Qiuyun Chen | Qing K. Wang

Cancer Nanomedicine: From Drug Delivery to Imaging

JOURNAL ARTICLE published 18 December 2013 in Science Translational Medicine

Authors: Edward Kai-Hua Chow | Dean Ho

Machine learning analysis of DNA methylation profiles distinguishes primary lung squamous cell carcinomas from head and neck metastases

JOURNAL ARTICLE published 11 September 2019 in Science Translational Medicine

Research funded by European Fund for Regional Development (EFRE) and the Federal State of Berlin (1303/2013) | Government of South Korea (2017-0-00451)

Authors: Philipp Jurmeister | Michael Bockmayr | Philipp Seegerer | Teresa Bockmayr | Denise Treue | Grégoire Montavon | Claudia Vollbrecht | Alexander Arnold | Daniel Teichmann | Keno Bressem | Ulrich Schüller | Maximilian von Laffert | Klaus-Robert Müller | David Capper | Frederick Klauschen

Tumor-derived circulating endothelial cell clusters in colorectal cancer

JOURNAL ARTICLE published 29 June 2016 in Science Translational Medicine

Authors: Igor Cima | Say Li Kong | Debarka Sengupta | Iain B. Tan | Wai Min Phyo | Daniel Lee | Min Hu | Ciprian Iliescu | Irina Alexander | Wei Lin Goh | Mehran Rahmani | Nur-Afidah Mohamed Suhaimi | Jess H. Vo | Joyce A. Tai | Joanna H. Tan | Clarinda Chua | Rachel Ten | Wan Jun Lim | Min Hoe Chew | Charlotte A. E. Hauser | Rob M. van Dam | Wei-Yen Lim | Shyam Prabhakar | Bing Lim | Poh Koon Koh | Paul Robson | Jackie Y. Ying | Axel M. Hillmer | Min-Han Tan

Rapamycin and Alzheimer’s disease: Time for a clinical trial?

JOURNAL ARTICLE published 23 January 2019 in Science Translational Medicine

Research funded by National Institute on Aging (P30AG013280) | National Institute on Aging (P30AG013319)

Authors: Matt Kaeberlein | Veronica Galvan

Mutant huntingtin and neurofilament light have distinct longitudinal dynamics in Huntington’s disease

JOURNAL ARTICLE published 16 December 2020 in Science Translational Medicine

Research funded by Wellcome (Wellcome Collaborative Award In Science 200181/Z/15/Z) | Wellcome (Wellcome/EPSRC Centre for Medical Engineering [WT 203148/Z/16/Z])

Authors: Filipe B. Rodrigues | Lauren M. Byrne | Rosanna Tortelli | Eileanoir B. Johnson | Peter A. Wijeratne | Marzena Arridge | Enrico De Vita | Naghmeh Ghazaleh | Richard Houghton | Hannah Furby | Daniel C. Alexander | Sarah J. Tabrizi | Scott Schobel | Rachael I. Scahill | Amanda Heslegrave | Henrik Zetterberg | Edward J. Wild

GDNF rescues the fate of neural progenitor grafts by attenuating Notch signals in the injured spinal cord in rodents

JOURNAL ARTICLE published 8 January 2020 in Science Translational Medicine

Authors: Mohamad Khazaei | Christopher S. Ahuja | Hiroaki Nakashima | Narihito Nagoshi | Lijun Li | Jian Wang | Jonathon Chio | Anna Badner | David Seligman | Ayaka Ichise | Shinsuke Shibata | Michael G. Fehlings

C9orf72 poly(GR) aggregation induces TDP-43 proteinopathy

JOURNAL ARTICLE published 2 September 2020 in Science Translational Medicine

Research funded by National Institute on Aging (R21AG065854) | National Institute of General Medical Sciences (R01GM099836) | National Institute of Neurological Disorders and Stroke (R35NS097273) | National Institute of Neurological Disorders and Stroke (P01NS084974) | National Institute of Neurological Disorders and Stroke (P01NS084974) | National Institute of Neurological Disorders and Stroke (P01NS099114) | National Institute of Neurological Disorders and Stroke (P01NS084974) | National Institute of Neurological Disorders and Stroke (P01NS084974) | National Institute of Neurological Disorders and Stroke (P01NS099114) | National Institute of Neurological Disorders and Stroke (R01NS088689)

Authors: Casey N. Cook | Yanwei Wu | Hana M. Odeh | Tania F. Gendron | Karen Jansen-West | Giulia del Rosso | Mei Yue | Peizhou Jiang | Edward Gomes | Jimei Tong | Lillian M. Daughrity | Nicole M. Avendano | Monica Castanedes-Casey | Wei Shao | Björn Oskarsson | Giulio S. Tomassy | Alexander McCampbell | Frank Rigo | Dennis W. Dickson | James Shorter | Yong-Jie Zhang | Leonard Petrucelli

PRMT5 control of cGAS/STING and NLRC5 pathways defines melanoma response to antitumor immunity

JOURNAL ARTICLE published 8 July 2020 in Science Translational Medicine

Research funded by National Institutes of Health (R35CA197465) | National Institutes of Health (R21CA198468) | National Institutes of Health (P30 CA030199) | Melanoma Research Alliance (509524) | DOD Peer Reviewed Cancer Research Program (CA1810216)

Authors: Hyungsoo Kim | Heejung Kim | Yongmei Feng | Yan Li | Hironari Tamiya | Stefania Tocci | Ze’ev A. Ronai

Glycine-based treatment ameliorates NAFLD by modulating fatty acid oxidation, glutathione synthesis, and the gut microbiome

JOURNAL ARTICLE published 2 December 2020 in Science Translational Medicine

Research funded by National Heart, Lung, and Blood Institute (HL137214, HL068878, HL134569) | National Heart, Lung, and Blood Institute (HL138139) | National Heart, Lung, and Blood Institute (HL123333) | National Heart, Lung, and Blood Institute (HL150233) | National Institute of Diabetes and Digestive and Kidney Diseases (DK106540) | American Heart Association (19POST34380224)

Authors: Oren Rom | Yuhao Liu | Zhipeng Liu | Ying Zhao | Jianfeng Wu | Alia Ghrayeb | Luis Villacorta | Yanbo Fan | Lin Chang | Lu Wang | Cai Liu | Dongshan Yang | Jun Song | Jason C. Rech | Yanhong Guo | Huilun Wang | Guizhen Zhao | Wenying Liang | Yui Koike | Haocheng Lu | Tomonari Koike | Tony Hayek | Subramaniam Pennathur | Chuanwu Xi | Bo Wen | Duxin Sun | Minerva T. Garcia-Barrio | Michael Aviram | Eyal Gottlieb | Inbal Mor | Wanqing Liu | Jifeng Zhang | Y. Eugene Chen

Analysis of recurrently protected genomic regions in cell-free DNA found in urine

JOURNAL ARTICLE published 17 February 2021 in Science Translational Medicine

Research funded by National Cancer Institute (U01CA243078) | National Cancer Institute (R01CA223481) | Science Foundation Arizona (BSP-0542-13)

Authors: Havell Markus | Jun Zhao | Tania Contente-Cuomo | Michelle D. Stephens | Elizabeth Raupach | Ahuva Odenheimer-Bergman | Sydney Connor | Bradon R. McDonald | Bethine Moore | Elizabeth Hutchins | Marissa McGilvrey | Michelina C. de la Maza | Kendall Van Keuren-Jensen | Patrick Pirrotte | Ajay Goel | Carlos Becerra | Daniel D. Von Hoff | Scott A. Celinski | Pooja Hingorani | Muhammed Murtaza

Restoring the natural tropism of AAV2 vectors for human liver

JOURNAL ARTICLE published 9 September 2020 in Science Translational Medicine

Research funded by National Science Foundation (DMS 1563234) | National Science Foundation (DMS 1563234) | Australian Research Council (DP150101253) | National Health and Medical Research Council (APP1156431) | National Health and Medical Research Council (APP1161583) | National Health and Medical Research Council (APP1108311) | National Health and Medical Research Council (APP1161583) | National Health and Medical Research Council (APP1156431) | Department of Science and Higher Education of Ministry of National Defense, Republic of Poland (k/10/8047/DNiSW/T – WIHE/3) | National Science Centre, Republic of Poland (UMO-2017/25/B/NZ1/02790)

Authors: Marti Cabanes-Creus | Claus V. Hallwirth | Adrian Westhaus | Boaz H. Ng | Sophia H.Y. Liao | Erhua Zhu | Renina Gale Navarro | Grober Baltazar | Matthieu Drouyer | Suzanne Scott | Grant J. Logan | Giorgia Santilli | Antonette Bennett | Samantha L. Ginn | Geoff McCaughan | Adrian J. Thrasher | Mavis Agbandje-McKenna | Ian E. Alexander | Leszek Lisowski

A Zebrafish Compound Screen Reveals Modulation of Neutrophil Reverse Migration as an Anti-Inflammatory Mechanism

JOURNAL ARTICLE published 26 February 2014 in Science Translational Medicine

Authors: Anne L. Robertson | Geoffrey R. Holmes | Aleksandra N. Bojarczuk | Joseph Burgon | Catherine A. Loynes | Myriam Chimen | Amy K. Sawtell | Bashar Hamza | Joseph Willson | Sarah R. Walmsley | Sean R. Anderson | Mark C. Coles | Stuart N. Farrow | Roberto Solari | Simon Jones | Lynne R. Prince | Daniel Irimia | G. Ed Rainger | Visakan Kadirkamanathan | Moira K. B. Whyte | Stephen A. Renshaw

Recombinant MG53 Protein Modulates Therapeutic Cell Membrane Repair in Treatment of Muscular Dystrophy

JOURNAL ARTICLE published 20 June 2012 in Science Translational Medicine

Authors: Noah Weisleder | Norio Takizawa | Peihui Lin | Xianhua Wang | Chunmei Cao | Yan Zhang | Tao Tan | Christopher Ferrante | Hua Zhu | Pin-Jung Chen | Rosalie Yan | Matthew Sterling | Xiaoli Zhao | Moonsun Hwang | Miyuki Takeshima | Chuanxi Cai | Heping Cheng | Hiroshi Takeshima | Rui-Ping Xiao | Jianjie Ma

Autoimmune receptor encephalitis in mice induced by active immunization with conformationally stabilized holoreceptors

JOURNAL ARTICLE published 10 July 2019 in Science Translational Medicine

Research funded by National Institutes of Health (NS080979) | National Institutes of Health (NS038631) | NINDS (P30NS061800)

Authors: Brian E. Jones | Kenneth R. Tovar | April Goehring | Farzad Jalali-Yazdi | Nana J. Okada | Eric Gouaux | Gary L. Westbrook

Single-cell profiling of human bone marrow progenitors reveals mechanisms of failing erythropoiesis in Diamond-Blackfan anemia

JOURNAL ARTICLE published 8 September 2021 in Science Translational Medicine

Authors: Deena Iskander | Guanlin Wang | Elisabeth F. Heuston | Chrysi Christodoulidou | Bethan Psaila | Kanagaraju Ponnusamy | Hongwei Ren | Zeinab Mokhtari | Mark Robinson | Aristeidis Chaidos | Pritesh Trivedi | Nikolaos Trasanidis | Alexia Katsarou | Richard Szydlo | Carmen G. Palii | Mehmood H. Zaidi | Qais Al-Oqaily | Valentina S. Caputo | Anindita Roy | Yvonne Harrington | Leena Karnik | Kikkeri Naresh | Adam J. Mead | Supat Thongjuea | Marjorie Brand | Josu de la Fuente | David M. Bodine | Irene Roberts | Anastasios Karadimitris

Activation of concurrent apoptosis and necroptosis by SMAC mimetics for the treatment of refractory and relapsed ALL

JOURNAL ARTICLE published 18 May 2016 in Science Translational Medicine

Research funded by Swiss Cancer League (KFS 3609-02-2015) | Swiss National Science Foundation (310030-133108) | Forschungskredit of the University of Zurich (FK-14-016)

Authors: Scott McComb | Júlia Aguadé-Gorgorió | Lena Harder | Blerim Marovca | Gunnar Cario | Cornelia Eckert | Martin Schrappe | Martin Stanulla | Arend von Stackelberg | Jean-Pierre Bourquin | Beat C. Bornhauser

Inhibiting the oncogenic translation program is an effective therapeutic strategy in multiple myeloma

JOURNAL ARTICLE published 10 May 2017 in Science Translational Medicine

Research funded by National Cancer Institute (award301791,R01 CA181683-01A1)

Authors: Salomon Manier | Daisy Huynh | Yu J. Shen | Jia Zhou | Timur Yusufzai | Karma Z. Salem | Richard Y. Ebright | Jiantao Shi | Jihye Park | Siobhan V. Glavey | William G. Devine | Chia-Jen Liu | Xavier Leleu | Bruno Quesnel | Catherine Roche-Lestienne | John K. Snyder | Lauren E. Brown | Nathanael Gray | James Bradner | Luke Whitesell | John A. Porco | Irene M. Ghobrial

Targeting pyrimidine synthesis accentuates molecular therapy response in glioblastoma stem cells

JOURNAL ARTICLE published 7 August 2019 in Science Translational Medicine

Research funded by National Cancer Institute (CA197718) | National Cancer Institute (CA154130) | National Cancer Institute (CA169117) | National Cancer Institute (CA171652) | National Cancer Institute (CA184090) | National Institute of Neurological Disorders and Stroke (NS087913) | National Institute of Neurological Disorders and Stroke (NS089272) | National Institute of Neurological Disorders and Stroke (NS091080) | National Institute of Neurological Disorders and Stroke (NS099175)

Authors: Xiuxing Wang | Kailin Yang | Qiulian Wu | Leo J. Y. Kim | Andrew R. Morton | Ryan C. Gimple | Briana C. Prager | Yu Shi | Wenchao Zhou | Shruti Bhargava | Zhe Zhu | Li Jiang | Weiwei Tao | Zhixin Qiu | Linjie Zhao | Guoxing Zhang | Xiqing Li | Sameer Agnihotri | Paul S. Mischel | Stephen C. Mack | Shideng Bao | Jeremy N. Rich

A long-duration dihydroorotate dehydrogenase inhibitor (DSM265) for prevention and treatment of malaria

JOURNAL ARTICLE published 15 July 2015 in Science Translational Medicine

Research funded by U.S. NIH (U01AI075594,R01AI103947) | Welch Foundation (I-1257) | Wellcome Trust (WT078285)

Authors: Margaret A. Phillips | Julie Lotharius | Kennan Marsh | John White | Anthony Dayan | Karen L. White | Jacqueline W. Njoroge | Farah El Mazouni | Yanbin Lao | Sreekanth Kokkonda | Diana R. Tomchick | Xiaoyi Deng | Trevor Laird | Sangeeta N. Bhatia | Sandra March | Caroline L. Ng | David A. Fidock | Sergio Wittlin | Maria Lafuente-Monasterio | Francisco Javier Gamo Benito | Laura Maria Sanz Alonso | Maria Santos Martinez | Maria Belen Jimenez-Diaz | Santiago Ferrer Bazaga | Iñigo Angulo-Barturen | John N. Haselden | James Louttit | Yi Cui | Arun Sridhar | Anna-Marie Zeeman | Clemens Kocken | Robert Sauerwein | Koen Dechering | Vicky M. Avery | Sandra Duffy | Michael Delves | Robert Sinden | Andrea Ruecker | Kristina S. Wickham | Rosemary Rochford | Janet Gahagen | Lalitha Iyer | Ed Riccio | Jon Mirsalis | Ian Bathhurst | Thomas Rueckle | Xavier Ding | Brice Campo | Didier Leroy | M. John Rogers | Pradipsinh K. Rathod | Jeremy N. Burrows | Susan A. Charman