Immune-mediated pathology in Duchenne muscular dystrophy.

abstract：:A clinical trial evaluating HIV vaccine therapy teaches us much about optimal design (Sneller et al, this issue). ...

journal_title：Science translational medicine

authors： Michael NL

更新日期：2017-12-06 00:00:00

abstract：:A set of principles is proposed for sponsors and developers of research computing applications that can increase the likelihood of successful adoption by researchers. ...

journal_title：Science translational medicine

authors： Masys DR,Harris PA,Fearn PA,Kohane IS

更新日期：2012-08-29 00:00:00

abstract：:Whole-genome sequencing of patient DNA can facilitate diagnosis of a disease, but its potential for guiding treatment has been under-realized. We interrogated the complete genome sequences of a 14-year-old fraternal twin pair diagnosed with dopa (3,4-dihydroxyphenylalanine)-responsive dystonia (DRD; Mendelian Inherita...

journal_title：Science translational medicine

authors： Bainbridge MN,Wiszniewski W,Murdock DR,Friedman J,Gonzaga-Jauregui C,Newsham I,Reid JG,Fink JK,Morgan MB,Gingras MC,Muzny DM,Hoang LD,Yousaf S,Lupski JR,Gibbs RA

更新日期：2011-06-15 00:00:00

abstract：:KRAS represents an excellent therapeutic target in lung cancer, the most commonly mutated form of which can now be blocked using KRAS-G12C mutant-specific inhibitory trial drugs. Lung adenocarcinoma cells harboring KRAS mutations have been shown previously to be selectively sensitive to inhibition of mitogen-activated...

journal_title：Science translational medicine

authors： Molina-Arcas M,Moore C,Rana S,van Maldegem F,Mugarza E,Romero-Clavijo P,Herbert E,Horswell S,Li LS,Janes MR,Hancock DC,Downward J

更新日期：2019-09-18 00:00:00

abstract：:Despite the remarkable enhancements in breast cancer classification and characterization, an exhaustive comprehension of the underlying carcinogenic processes and an accurate prediction of its clinical behavior are yet to be achieved. On the wave of recent scientific advances and clinical findings, new cutting-edge te...

journal_title：Science translational medicine

authors： Reis-Filho JS,Weigelt B,Fumagalli D,Sotiriou C

更新日期：2010-09-01 00:00:00

abstract：:People with autism spectrum disorder (ASD) are characterized by deficits in social interaction, language, and behavioral flexibility. Rare mutations and copy number variations have been identified in individuals with ASD, but in most patients, the causal variants remain unknown. A genome-wide association study (GWAS),...

journal_title：Science translational medicine

authors： Kerin T,Ramanathan A,Rivas K,Grepo N,Coetzee GA,Campbell DB

更新日期：2012-04-04 00:00:00

abstract：:Therapeutic intravesical instillation of bacillus Calmette-Guérin (BCG) is effective at triggering inflammation and eliciting successful tumor immunity in patients with non-muscle invasive bladder cancer, with 50 to 70% clinical response. Therapeutic success relies on repeated instillations of live BCG administered as...

journal_title：Science translational medicine

authors： Biot C,Rentsch CA,Gsponer JR,Birkhäuser FD,Jusforgues-Saklani H,Lemaître F,Auriau C,Bachmann A,Bousso P,Demangel C,Peduto L,Thalmann GN,Albert ML

更新日期：2012-06-06 00:00:00

abstract：:Functional magnetic resonance imaging offers an unprecedented opportunity to evaluate and compare drug effects on human brain activity and to provide systems-level predictions for how new drugs for chronic pain will affect the brain, thus accelerating drug discovery and repurposing (Duff et al., this issue). ...

journal_title：Science translational medicine

authors： Wager TD,Woo CW

更新日期：2015-02-11 00:00:00

abstract：:Exposure to tobacco smoke activates innate and adaptive immune responses that in long-term smokers have been linked to diseases of the lungs, cardiovascular system, joints, and other organs. The destruction of lung tissue that underlies smoking-induced emphysema has been associated with T helper 1 cells that recognize...

journal_title：Science translational medicine

authors： Shan M,Cheng HF,Song LZ,Roberts L,Green L,Hacken-Bitar J,Huh J,Bakaeen F,Coxson HO,Storness-Bliss C,Ramchandani M,Lee SH,Corry DB,Kheradmand F

更新日期：2009-10-28 00:00:00

abstract：:Motor neuron-specific microRNA-218 (miR-218) has recently received attention because of its roles in mouse development. However, miR-218 relevance to human motor neuron disease was not yet explored. Here, we demonstrate by neuropathology that miR-218 is abundant in healthy human motor neurons. However, in amyotrophic ...

journal_title：Science translational medicine

authors： Reichenstein I,Eitan C,Diaz-Garcia S,Haim G,Magen I,Siany A,Hoye ML,Rivkin N,Olender T,Toth B,Ravid R,Mandelbaum AD,Yanowski E,Liang J,Rymer JK,Levy R,Beck G,Ainbinder E,Farhan SMK,Lennox KA,Bode NM,Behlke MA,

更新日期：2019-12-18 00:00:00

abstract：:The four dengue virus serotypes (DENV1 to DENV4) are mosquito-borne flaviviruses that cause up to ~100 million cases of dengue annually worldwide. Severe disease is thought to result from immunopathogenic processes involving serotype cross-reactive antibodies and T cells that together induce vasoactive cytokines, caus...

journal_title：Science translational medicine

authors： Beatty PR,Puerta-Guardo H,Killingbeck SS,Glasner DR,Hopkins K,Harris E

更新日期：2015-09-09 00:00:00

abstract：:Alzheimer's disease (AD) is hypothesized to be caused by an overproduction or reduced clearance of amyloid-β (Aβ) peptide. Autosomal dominant AD (ADAD) caused by mutations in the presenilin (PSEN) gene have been postulated to result from increased production of Aβ42 compared to Aβ40 in the central nervous system (CNS)...

journal_title：Science translational medicine

authors： Potter R,Patterson BW,Elbert DL,Ovod V,Kasten T,Sigurdson W,Mawuenyega K,Blazey T,Goate A,Chott R,Yarasheski KE,Holtzman DM,Morris JC,Benzinger TL,Bateman RJ

更新日期：2013-06-12 00:00:00

abstract：:Tuberculous meningitis (TBM) is a devastating form of tuberculosis (TB), and key TB antimicrobials, including rifampin, have restricted brain penetration. A lack of reliable data on intralesional drug biodistribution in infected tissues has limited pharmacokinetic (PK) modeling efforts to optimize TBM treatments. Curr...

journal_title：Science translational medicine

authors： Tucker EW,Guglieri-Lopez B,Ordonez AA,Ritchie B,Klunk MH,Sharma R,Chang YS,Sanchez-Bautista J,Frey S,Lodge MA,Rowe SP,Holt DP,Gobburu JVS,Peloquin CA,Mathews WB,Dannals RF,Pardo CA,Kannan S,Ivaturi VD,Jain SK

更新日期：2018-12-05 00:00:00

abstract：:Myofibroblast-derived αvβ1 integrin has emerged as a critical mediator of transforming growth factor-β activation in experimental models of tissue fibrosis (Reed et al., this issue). ...

journal_title：Science translational medicine

authors： Dietz HC

更新日期：2015-05-20 00:00:00

abstract：:Prenatal and postnatal treatment with a CFTR modifier attenuates pathological changes in a ferret model of cystic fibrosis (Sun et al., this issue). ...

journal_title：Science translational medicine

authors： Ferkol TW

更新日期：2019-03-27 00:00:00

abstract：:We outline the fundamental properties of a highly participatory rapid learning system that can be developed in part from meaningful use of electronic health records (EHRs). Future widespread adoption of EHRs will make increasing amounts of medical information available in computable form. Secured and trusted use of th...

journal_title：Science translational medicine

authors： Friedman CP,Wong AK,Blumenthal D

更新日期：2010-11-10 00:00:00

abstract：:MG53 promotes sarcolemmal repair in the mdx mouse model of Duchenne muscular dystrophy (Weisleder et al., this issue) and identifies a new protein therapeutic for muscle disease. ...

journal_title：Science translational medicine

authors： Burkin DJ,Wuebbles RD

更新日期：2012-06-20 00:00:00

abstract：:Inhaled drugs offer advantages, such as rapid onset of action, but require formulations and delivery systems that reproducibly and conveniently administer the drug. CVT-301 is a powder formulation of levodopa delivered by a breath-actuated inhaler that has been developed for treating OFF episodes (motor fluctuations b...

journal_title：Science translational medicine

authors： Lipp MM,Batycky R,Moore J,Leinonen M,Freed MI

更新日期：2016-10-12 00:00:00

abstract：:The Point-Person Project, a child-health research initiative, enables rapid response to opportunities for participation in multicenter pediatric clinical trials. ...

journal_title：Science translational medicine

authors： Davis JM,Smoyer WE,Connor EM,Huskins WC,Pastern C,Purucker M,Schrader EC,Jackson CR,Kaskel FJ,Hirschfeld S

更新日期：2015-03-18 00:00:00

abstract：:A rare neurodevelopmental disorder in the Old Order Mennonite population called PMSE (polyhydramnios, megalencephaly, and symptomatic epilepsy syndrome; also called Pretzel syndrome) is characterized by infantile-onset epilepsy, neurocognitive delay, craniofacial dysmorphism, and histopathological evidence of heteroto...

journal_title：Science translational medicine

authors： Parker WE,Orlova KA,Parker WH,Birnbaum JF,Krymskaya VP,Goncharov DA,Baybis M,Helfferich J,Okochi K,Strauss KA,Crino PB

更新日期：2013-04-24 00:00:00

abstract：:The invasive nature of glioblastoma (GBM) represents a major clinical challenge contributing to poor outcomes. Invasion of GBM into healthy tissue restricts chemotherapeutic access and complicates surgical resection. Here, we test the hypothesis that an effective anti-invasive agent can "contain" GBM and increase the ...

journal_title：Science translational medicine

authors： Munson JM,Fried L,Rowson SA,Bonner MY,Karumbaiah L,Diaz B,Courtneidge SA,Knaus UG,Brat DJ,Arbiser JL,Bellamkonda RV

更新日期：2012-03-28 00:00:00

abstract：:Tumor escape from immune-mediated destruction has been associated with immunosuppressive mechanisms that inhibit T cell activation. Although evidence for an active immune response, including infiltration with CD8(+) T cells, can be found in a subset of patients, those tumors are nonetheless not immunologically rejecte...

journal_title：Science translational medicine

authors： Spranger S,Spaapen RM,Zha Y,Williams J,Meng Y,Ha TT,Gajewski TF

更新日期：2013-08-28 00:00:00

abstract：:The clinical application of cytokine therapies for cancer treatment remains limited due to severe adverse reactions and insufficient therapeutic effects. Although cytokine localization by intratumoral administration could address both issues, the rapid escape of soluble cytokines from the tumor invariably subverts thi...

journal_title：Science translational medicine

authors： Momin N,Mehta NK,Bennett NR,Ma L,Palmeri JR,Chinn MM,Lutz EA,Kang B,Irvine DJ,Spranger S,Wittrup KD

更新日期：2019-06-26 00:00:00

abstract：:The role of neuronal Toll-like receptor 4 (TLR4) in nerve injury is being pursued actively. However, the endogenous activation of neuronal TLR4 during neuroinflammation, in absence of the participation of glial TLR4, remains elusive. Here, we identified lysozyme as an endogenous activator of neuronal TLR4 signaling du...

journal_title：Science translational medicine

authors： Yadav S,Surolia A

更新日期：2019-08-07 00:00:00

abstract：:Fibroblasts are primary cellular protagonists of wound healing. They also exhibit circadian timekeeping, which imparts an approximately 24-hour rhythm to their biological function. We interrogated the functional consequences of the cell-autonomous clockwork in fibroblasts using a proteome-wide screen for rhythmically ...

journal_title：Science translational medicine

authors： Hoyle NP,Seinkmane E,Putker M,Feeney KA,Krogager TP,Chesham JE,Bray LK,Thomas JM,Dunn K,Blaikley J,O'Neill JS

更新日期：2017-11-08 00:00:00

abstract：:Pharmacokinetic and pharmacodynamic models estimate the potency of antiviral agents but do not capture viral and immunologic factors that drive the natural dynamics of infection. We designed a mathematical model that synthesizes pharmacokinetics, pharmacodynamics, and viral pathogenesis concepts to simulate the activi...

journal_title：Science translational medicine

authors： Schiffer JT,Swan DA,Magaret A,Corey L,Wald A,Ossig J,Ruebsamen-Schaeff H,Stoelben S,Timmler B,Zimmermann H,Melhem MR,Van Wart SA,Rubino CM,Birkmann A

更新日期：2016-02-03 00:00:00

abstract：:New insights into the mechanism of action of ebselen, a small-molecule antivirulence agent that reduces disease pathology in a mouse model of Clostridium difficile infection, suggest a different molecular target may be responsible for its efficacy. ...

journal_title：Science translational medicine

authors： Beilhartz GL,Tam J,Zhang Z,Melnyk RA

更新日期：2016-12-21 00:00:00

abstract：:Pluripotent stem cells are a scalable source of pancreatic cells for transplantation into patients with diabetes. Here, we describe how the field is gaining momentum toward a β cell replacement therapy. ...

journal_title：Science translational medicine

authors： Bruin JE,Rezania A,Kieffer TJ

更新日期：2015-12-02 00:00:00

abstract：:Low-grade polymicrobial infection induced by cecal ligation and puncture is lethal in heme oxygenase-1-deficient mice (Hmox1(-/-)), but not in wild-type (Hmox1(+/+)) mice. Here we demonstrate that the protective effect of this heme-catabolizing enzyme relies on its ability to prevent tissue damage caused by the circul...

journal_title：Science translational medicine

authors： Larsen R,Gozzelino R,Jeney V,Tokaji L,Bozza FA,Japiassú AM,Bonaparte D,Cavalcante MM,Chora A,Ferreira A,Marguti I,Cardoso S,Sepúlveda N,Smith A,Soares MP

更新日期：2010-09-29 00:00:00

abstract：:As part of the global effort toward malaria eradication, phenotypic whole-cell screening revealed the 2-aminopyridine class of small molecules as a good starting point to develop new antimalarial drugs. Stemming from this series, we found that the derivative, MMV390048, lacked cross-resistance with current drugs used ...

journal_title：Science translational medicine

authors： Paquet T,Le Manach C,Cabrera DG,Younis Y,Henrich PP,Abraham TS,Lee MCS,Basak R,Ghidelli-Disse S,Lafuente-Monasterio MJ,Bantscheff M,Ruecker A,Blagborough AM,Zakutansky SE,Zeeman AM,White KL,Shackleford DM,Mannila J,Mo

更新日期：2017-04-26 00:00:00