Catastrophic childhood epilepsy: a recent convergence of basic and clinical neuroscience.

abstract：:SORLA/SORL1 is a unique neuronal sorting receptor for the amyloid precursor protein that has been causally implicated in both sporadic and autosomal dominant familial forms of Alzheimer's disease (AD). Brain concentrations of SORLA are inversely correlated with amyloid-β (Aβ) in mouse models and AD patients, suggestin...

journal_title：Science translational medicine

authors： Caglayan S,Takagi-Niidome S,Liao F,Carlo AS,Schmidt V,Burgert T,Kitago Y,Füchtbauer EM,Füchtbauer A,Holtzman DM,Takagi J,Willnow TE

更新日期：2014-02-12 00:00:00

abstract：:The limited predictability of phase II biomarkers for atherosclerosis outcomes in phase III studies stands in contrast to the number and varied types of biomarkers--soluble, imaging, and functional--that have been used in a diverse array of trials. Although collectively abundant, these biomarker data exist in a fragme...

journal_title：Science translational medicine

authors： Fryburg DA,Vassileva MT

更新日期：2011-03-02 00:00:00

abstract：:The Commentary of Mills and Sykes in Science Translational Medicine presented their thesis on the advent of high-throughput technologies and the dangers they may represent for the future of biomedical research. In response, we argue that true progress on the diagnosis and treatment of common human diseases will requir...

journal_title：Science translational medicine

authors： Lamb JR,Gibson NW

更新日期：2010-03-31 00:00:00

abstract：:Children who survive preterm birth exhibit persistent unexplained disturbances in cerebral cortical growth with associated cognitive and learning disabilities. The mechanisms underlying these deficits remain elusive. We used ex vivo diffusion magnetic resonance imaging to demonstrate in a preterm large-animal model th...

journal_title：Science translational medicine

authors： Dean JM,McClendon E,Hansen K,Azimi-Zonooz A,Chen K,Riddle A,Gong X,Sharifnia E,Hagen M,Ahmad T,Leigland LA,Hohimer AR,Kroenke CD,Back SA

更新日期：2013-01-16 00:00:00

abstract：:Rapid advances in the forward engineering of genetic circuitry in living cells has positioned synthetic biology as a potential means to solve numerous biomedical problems, including disease diagnosis and therapy. One challenge in exploiting synthetic biology for translational applications is to engineer microbes that ...

journal_title：Science translational medicine

authors： Danino T,Prindle A,Kwong GA,Skalak M,Li H,Allen K,Hasty J,Bhatia SN

更新日期：2015-05-27 00:00:00

abstract：:Detection of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infections to date has relied heavily on reverse transcription polymerase chain reaction testing. However, limited test availability, high false-negative rates, and the existence of asymptomatic or subclinical infections have resulted in an unde...

journal_title：Science translational medicine

authors： Silverman JD,Hupert N,Washburne AD

更新日期：2020-07-29 00:00:00

abstract：:Measles virus is a highly infectious morbillivirus responsible for major morbidity and mortality in unvaccinated humans. The related, zoonotic canine distemper virus (CDV) induces morbillivirus disease in ferrets with 100% lethality. We report an orally available, shelf-stable pan-morbillivirus inhibitor that targets ...

journal_title：Science translational medicine

authors： Krumm SA,Yan D,Hovingh ES,Evers TJ,Enkirch T,Reddy GP,Sun A,Saindane MT,Arrendale RF,Painter G,Liotta DC,Natchus MG,von Messling V,Plemper RK

更新日期：2014-04-16 00:00:00

abstract：:Pathological angiogenesis is the hallmark of diseases such as cancer and retinopathies. Although tissue hypoxia and inflammation are recognized as central drivers of vessel growth, relatively little is known about the process that bridges the two. In a mouse model of ischemic retinopathy, we found that hypoxic regions...

journal_title：Science translational medicine

authors： Oubaha M,Miloudi K,Dejda A,Guber V,Mawambo G,Germain MA,Bourdel G,Popovic N,Rezende FA,Kaufman RJ,Mallette FA,Sapieha P

更新日期：2016-10-26 00:00:00

abstract：:Most cancer deaths arise from metastases as a result of circulating tumor cells (CTCs) spreading from the primary tumor to vital organs. Despite progress in cancer prognosis, the role of CTCs in early disease diagnosis is unclear because of the low sensitivity of CTC assays. We demonstrate the high sensitivity of the ...

journal_title：Science translational medicine

authors： Galanzha EI,Menyaev YA,Yadem AC,Sarimollaoglu M,Juratli MA,Nedosekin DA,Foster SR,Jamshidi-Parsian A,Siegel ER,Makhoul I,Hutchins LF,Suen JY,Zharov VP

更新日期：2019-06-12 00:00:00

abstract：:Sunitinib malate is a multitargeted receptor tyrosine kinase inhibitor used in the treatment of human malignancies. A substantial number of sunitinib-treated patients develop cardiac dysfunction, but the mechanism of sunitinib-induced cardiotoxicity is poorly understood. We show that mice treated with sunitinib develo...

journal_title：Science translational medicine

authors： Chintalgattu V,Rees ML,Culver JC,Goel A,Jiffar T,Zhang J,Dunner K Jr,Pati S,Bankson JA,Pasqualini R,Arap W,Bryan NS,Taegtmeyer H,Langley RR,Yao H,Kupferman ME,Entman ML,Dickinson ME,Khakoo AY

更新日期：2013-05-29 00:00:00

abstract：:Neutralizing antibodies to the V2 apex antigenic region of the HIV-1 envelope (Env) trimer are among the most prevalent cross-reactive antibodies elicited by natural infection. Two recently described V2-specific antibodies, PGDM1400 and CAP256-VRC26.25, have demonstrated exquisite potency and neutralization breadth ag...

journal_title：Science translational medicine

authors： Julg B,Tartaglia LJ,Keele BF,Wagh K,Pegu A,Sok D,Abbink P,Schmidt SD,Wang K,Chen X,Joyce MG,Georgiev IS,Choe M,Kwong PD,Doria-Rose NA,Le K,Louder MK,Bailer RT,Moore PL,Korber B,Seaman MS,Abdool Karim SS,Morris

更新日期：2017-09-06 00:00:00

abstract：:Trauma-induced critical illness is driven by acute inflammation, and elevated systemic interleukin-6 (IL-6) after trauma is a biomarker of adverse outcomes. We constructed a multicompartment, ordinary differential equation model that represents a virtual trauma patient. Individual-specific variants of this model repro...

journal_title：Science translational medicine

authors： Brown D,Namas RA,Almahmoud K,Zaaqoq A,Sarkar J,Barclay DA,Yin J,Ghuma A,Abboud A,Constantine G,Nieman G,Zamora R,Chang SC,Billiar TR,Vodovotz Y

更新日期：2015-04-29 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：:EphB receptors regulate the proliferation and positioning of intestinal stem and progenitor cells. In addition, they can act as tumor promoters for adenoma development but suppress progression to invasive carcinoma. We used imatinib to abrogate Abl kinase activity in Apc(Min/+) mice and in mice with LGR5(+) stem cells...

journal_title：Science translational medicine

authors： Kundu P,Genander M,Strååt K,Classon J,Ridgway RA,Tan EH,Björk J,Martling A,van Es J,Sansom OJ,Clevers H,Pettersson S,Frisén J

更新日期：2015-04-01 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：:About one-third of people in the developed world will undergo cataract surgery in their lifetime. Although marked improvements in surgical technique have occurred since the development of the current approach to lens replacement in the late 1960s and early 1970s, some critical steps of the procedure can still only be ...

journal_title：Science translational medicine

authors： Palanker DV,Blumenkranz MS,Andersen D,Wiltberger M,Marcellino G,Gooding P,Angeley D,Schuele G,Woodley B,Simoneau M,Friedman NJ,Seibel B,Batlle J,Feliz R,Talamo J,Culbertson W

更新日期：2010-11-17 00:00:00

abstract：:Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease caused by loss of activity of α-l-iduronidase and attendant accumulation of the glycosaminoglycans dermatan sulfate and heparan sulfate. Current treatments are suboptimal and do not address residual disease including corneal clouding, skeletal deformi...

journal_title：Science translational medicine

authors： Dierenfeld AD,McEntee MF,Vogler CA,Vite CH,Chen AH,Passage M,Le S,Shah S,Jens JK,Snella EM,Kline KL,Parkes JD,Ware WA,Moran LE,Fales-Williams AJ,Wengert JA,Whitley RD,Betts DM,Boal AM,Riedesel EA,Gross W,Ellinwo

更新日期：2010-12-01 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

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：:Nipah virus is an emerging pathogen in the Paramyxoviridae family. Upon transmission of Nipah virus from its natural reservoir, Pteropus spp. fruit bats, to humans, it causes respiratory and neurological disease with a case-fatality rate about 70%. Human-to-human transmission has been observed during Nipah virus outbr...

journal_title：Science translational medicine

authors： Lo MK,Feldmann F,Gary JM,Jordan R,Bannister R,Cronin J,Patel NR,Klena JD,Nichol ST,Cihlar T,Zaki SR,Feldmann H,Spiropoulou CF,de Wit E

更新日期：2019-05-29 00:00:00

abstract：:Stimulator of interferon genes (STING) is a cytosolic receptor that senses both exogenous and endogenous cytosolic cyclic dinucleotides (CDNs), activating TBK1/IRF3 (interferon regulatory factor 3), NF-κB (nuclear factor κB), and STAT6 (signal transducer and activator of transcription 6) signaling pathways to induce r...

journal_title：Science translational medicine

authors： Fu J,Kanne DB,Leong M,Glickman LH,McWhirter SM,Lemmens E,Mechette K,Leong JJ,Lauer P,Liu W,Sivick KE,Zeng Q,Soares KC,Zheng L,Portnoy DA,Woodward JJ,Pardoll DM,Dubensky TW Jr,Kim Y

更新日期：2015-04-15 00:00:00

abstract：:Sustained viremia after acute HIV infection is associated with profound CD4+ T cell loss and exhaustion of HIV-specific CD8+ T cell responses. To determine the impact of combination antiretroviral therapy (cART) on these processes, we examined the evolution of immune responses in acutely infected individuals initiatin...

journal_title：Science translational medicine

authors： Ndhlovu ZM,Kazer SW,Nkosi T,Ogunshola F,Muema DM,Anmole G,Swann SA,Moodley A,Dong K,Reddy T,Brockman MA,Shalek AK,Ndung'u T,Walker BD

更新日期：2019-05-22 00:00:00

abstract：:Canavan disease is a hereditary leukodystrophy caused by mutations in the aspartoacylase gene (ASPA), leading to loss of enzyme activity and increased concentrations of the substrate N-acetyl-aspartate (NAA) in the brain. Accumulation of NAA results in spongiform degeneration of white matter and severe impairment of p...

journal_title：Science translational medicine

authors： Leone P,Shera D,McPhee SW,Francis JS,Kolodny EH,Bilaniuk LT,Wang DJ,Assadi M,Goldfarb O,Goldman HW,Freese A,Young D,During MJ,Samulski RJ,Janson CG

更新日期：2012-12-19 00:00:00

abstract：:Limited treatment options exist for patients who suffer from a painful bladder condition known as interstitial cystitis/bladder pain syndrome (IC/BPS). Whether given systemically (orally) or by short-duration (1 to 2 hours) exposure via intravesical instillation, therapeutic agents have exhibited poor efficacy because...

journal_title：Science translational medicine

authors： Nickel JC,Jain P,Shore N,Anderson J,Giesing D,Lee H,Kim G,Daniel K,White S,Larrivee-Elkins C,Lekstrom-Himes J,Cima M

更新日期：2012-07-18 00:00:00

abstract：:DEL-1 (developmental endothelial locus-1) is an endothelial cell-secreted protein that regulates LFA-1 (lymphocyte function-associated antigen-1) integrin-dependent leukocyte recruitment and inflammation in various tissues. We identified a novel regulatory mechanism of DEL-1 in osteoclast biology. Specifically, we sho...

journal_title：Science translational medicine

authors： Shin J,Maekawa T,Abe T,Hajishengallis E,Hosur K,Pyaram K,Mitroulis I,Chavakis T,Hajishengallis G

更新日期：2015-09-30 00:00:00

abstract：:Atherosclerosis is the cause of morbiditiy for 70% of patients with type 2 diabetes. In both of these diseases, a protein complex known as the inflammasome is stimulated to activate interleukin-1β (IL-1β) and IL-18, which are pathogenic inflammatory cytokines. Triggers for the inflammasome are obesity-related factors,...

journal_title：Science translational medicine

authors： Masters SL,Latz E,O'Neill LA

更新日期：2011-05-04 00:00:00

abstract：:Research standards deviate in genetic versus nongenetic epidemiology. Besides some immutable differences, such as the correlation pattern between variables, these divergent research standards can converge considerably. Current research designs that dissociate genetic and nongenetic measurements are reaching their limi...

journal_title：Science translational medicine

authors： Ioannidis JP,Loy EY,Poulton R,Chia KS

更新日期：2009-11-18 00:00:00

abstract：:Obesity increases the risk of adverse outcomes during acute critical illnesses such as burns, severe trauma, and acute pancreatitis. Although individuals with more body fat and higher serum cytokines and lipase are more likely to experience problems, the roles that these characteristics play are not clear. We used sev...

journal_title：Science translational medicine

authors： Navina S,Acharya C,DeLany JP,Orlichenko LS,Baty CJ,Shiva SS,Durgampudi C,Karlsson JM,Lee K,Bae KT,Furlan A,Behari J,Liu S,McHale T,Nichols L,Papachristou GI,Yadav D,Singh VP

更新日期：2011-11-02 00:00:00

abstract：:Two U.S. national initiatives have stated emphatically that people will be, not patients, but partners in clinical research. ...

journal_title：Science translational medicine

authors： Terry SF

更新日期：2017-01-04 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