A bioengineered retinal pigment epithelial monolayer for advanced, dry age-related macular degeneration.

abstract：:Biallelic mutations in ABCC6 cause pseudoxanthoma elasticum (PXE), a disease characterized by calcification in the skin, eyes, and blood vessels. The function of ATP-binding cassette C6 (ABCC6) and the pathogenesis of PXE remain unclear. We used mouse models and patient fibroblasts to demonstrate genetic interaction a...

journal_title：Science translational medicine

authors： Ziegler SG,Ferreira CR,MacFarlane EG,Riddle RC,Tomlinson RE,Chew EY,Martin L,Ma CT,Sergienko E,Pinkerton AB,Millán JL,Gahl WA,Dietz HC

更新日期：2017-06-07 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：:Rapidly spreading antibiotic resistance and the low discovery rate of new antimicrobial compounds demand more effective strategies for early drug discovery. One bottleneck in the drug discovery pipeline is the identification of the modes of action (MoAs) of new compounds. We have developed a rapid systematic metabolom...

journal_title：Science translational medicine

authors： Zampieri M,Szappanos B,Buchieri MV,Trauner A,Piazza I,Picotti P,Gagneux S,Borrell S,Gicquel B,Lelievre J,Papp B,Sauer U

更新日期：2018-02-21 00:00:00

abstract：:A primary barrier to the success of T cell-recruiting bispecific antibodies in the treatment of solid tumors is the lack of tumor-specific targets, resulting in on-target off-tumor adverse effects from T cell autoreactivity to target-expressing organs. To overcome this, we developed an anti-HER2/CD3 T cell-dependent b...

journal_title：Science translational medicine

authors： Slaga D,Ellerman D,Lombana TN,Vij R,Li J,Hristopoulos M,Clark R,Johnston J,Shelton A,Mai E,Gadkar K,Lo AA,Koerber JT,Totpal K,Prell R,Lee G,Spiess C,Junttila TT

更新日期：2018-10-17 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：:Metabolic reprogramming is linked to cancer cell growth and proliferation, metastasis, and therapeutic resistance in a multitude of cancers. Targeting dysregulated metabolic pathways to overcome resistance, an urgent clinical need in all relapsed/refractory cancers, remains difficult. Through genomic analyses of clini...

journal_title：Science translational medicine

authors： Zhang L,Yao Y,Zhang S,Liu Y,Guo H,Ahmed M,Bell T,Zhang H,Han G,Lorence E,Badillo M,Zhou S,Sun Y,Di Francesco ME,Feng N,Haun R,Lan R,Mackintosh SG,Mao X,Song X,Zhang J,Pham LV,Lorenzi PL,Marszalek J,Heffern

更新日期：2019-05-08 00:00:00

abstract：:Despite the very low risk-to-benefit ratio of vaccines, fear of negative side effects has discouraged many people from getting vaccinated, resulting in reemergence of previously controlled diseases such as measles, pertussis, and diphtheria. Part of this fear stems from the lack of public awareness of the many preclin...

journal_title：Science translational medicine

authors： Ahmed SS,Plotkin SA,Black S,Coffman RL

更新日期：2011-07-27 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：:Direct-to-consumer (DTC) advertising of suspect goods and services has burgeoned because of the Internet. Despite very limited approval for use, DTC stem cell-marketed "treatments" have emerged for an array of conditions, creating global public health and safety risks. However, it remains unclear whether such use of s...

journal_title：Science translational medicine

authors： Liang BA,Mackey TK

更新日期：2012-09-12 00:00:00

abstract：:In humans, exposure to aristolochic acid (AA) is associated with urothelial carcinoma of the upper urinary tract (UTUC). Exome sequencing of UTUCs from 19 individuals with documented exposure to AA revealed a remarkably large number of somatic mutations and an unusual mutational signature attributable to AA. Most of t...

journal_title：Science translational medicine

authors： Hoang ML,Chen CH,Sidorenko VS,He J,Dickman KG,Yun BH,Moriya M,Niknafs N,Douville C,Karchin R,Turesky RJ,Pu YS,Vogelstein B,Papadopoulos N,Grollman AP,Kinzler KW,Rosenquist TA

更新日期：2013-08-07 00:00:00

abstract：:Patients undergoing surgical resection of primary breast tumors confront a risk for metastatic recurrence that peaks sharply 12 to 18 months after surgery. The cause of early metastatic relapse in breast cancer has long been debated, with many ascribing these relapses to the natural progression of the disease. Others ...

journal_title：Science translational medicine

authors： Krall JA,Reinhardt F,Mercury OA,Pattabiraman DR,Brooks MW,Dougan M,Lambert AW,Bierie B,Ploegh HL,Dougan SK,Weinberg RA

更新日期：2018-04-11 00:00:00

abstract：:Reactivation of fetal hemoglobin (HbF) is being pursued as a treatment strategy for hemoglobinopathies. Here, we evaluated the therapeutic potential of hematopoietic stem and progenitor cells (HSPCs) edited with the CRISPR-Cas9 nuclease platform to recapitulate naturally occurring mutations identified in individuals w...

journal_title：Science translational medicine

authors： Humbert O,Radtke S,Samuelson C,Carrillo RR,Perez AM,Reddy SS,Lux C,Pattabhi S,Schefter LE,Negre O,Lee CM,Bao G,Adair JE,Peterson CW,Rawlings DJ,Scharenberg AM,Kiem HP

更新日期：2019-07-31 00:00:00

abstract：:Regular physical activity and especially aerobic exercise are associated with reduced risk of disease and enhanced longevity, but the molecular mechanisms of these health benefits remain obscure. A comprehensive metabolomic approach was used to characterize the changes in blood levels of >200 metabolites upon vigorous...

journal_title：Science translational medicine

authors： Burke MF,Dunbar RL,Rader DJ

更新日期：2010-07-21 00:00:00

abstract：:Acquired lymphedema is a cancer sequela and a global health problem currently lacking pharmacologic therapy. We have previously demonstrated that ketoprofen, an anti-inflammatory agent with dual 5-lipoxygenase and cyclooxygenase inhibitory properties, effectively reverses histopathology in experimental lymphedema. We ...

journal_title：Science translational medicine

authors： Tian W,Rockson SG,Jiang X,Kim J,Begaye A,Shuffle EM,Tu AB,Cribb M,Nepiyushchikh Z,Feroze AH,Zamanian RT,Dhillon GS,Voelkel NF,Peters-Golden M,Kitajewski J,Dixon JB,Nicolls MR

更新日期：2017-05-10 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：:As the prevalence of Alzheimer's disease (AD) grows, so do the costs it imposes on society. Scientific, clinical, and financial interests have focused current drug discovery efforts largely on the single biological pathway that leads to amyloid deposition. This effort has resulted in slow progress and disappointing ou...

journal_title：Science translational medicine

authors： Lo AW,Ho C,Cummings J,Kosik KS

更新日期：2014-06-18 00:00:00

abstract：:Amine-containing polymers immobilized on mesh and placed at trauma sites scavenge biomolecules that initiate a damaging immune response. ...

journal_title：Science translational medicine

authors： Ulery BD

更新日期：2017-02-22 00:00:00

abstract：:Retinal prosthetic implants are the only approved treatment for retinitis pigmentosa, a disease of the eye that causes blindness through gradual degeneration of photoreceptors. An array of microelectrodes triggered by input from a camera stimulates surviving retinal neurons, with each electrode acting as a pixel. Unin...

journal_title：Science translational medicine

authors： Weitz AC,Nanduri D,Behrend MR,Gonzalez-Calle A,Greenberg RJ,Humayun MS,Chow RH,Weiland JD

更新日期：2015-12-16 00:00:00

abstract：:The etiology of major depression remains unknown, but dysfunction of serotonergic signaling has long been implicated in the pathophysiology of this disorder. p11 is an S100 family member recently identified as a serotonin 1B [5-hydroxytryptamine 1B (5-HT(1B))] and serotonin 4 (5-HT(4)) receptor-binding protein. Mutant...

journal_title：Science translational medicine

authors： Alexander B,Warner-Schmidt J,Eriksson T,Tamminga C,Arango-Lievano M,Ghose S,Vernov M,Stavarache M,Musatov S,Flajolet M,Svenningsson P,Greengard P,Kaplitt MG

更新日期：2010-10-20 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：: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：:Down syndrome (DS) is among the most frequent genetic causes of intellectual disability, and ameliorating this deficit is a major goal in support of people with trisomy 21. The Ts65Dn mouse recapitulates some major brain structural and behavioral phenotypes of DS, including reduced size and cellularity of the cerebell...

journal_title：Science translational medicine

authors： Das I,Park JM,Shin JH,Jeon SK,Lorenzi H,Linden DJ,Worley PF,Reeves RH

更新日期：2013-09-04 00:00:00

abstract：:Physiological data are routinely recorded in intensive care, but their use for rapid assessment of illness severity or long-term morbidity prediction has been limited. We developed a physiological assessment score for preterm newborns, akin to an electronic Apgar score, based on standard signals recorded noninvasively...

journal_title：Science translational medicine

authors： Saria S,Rajani AK,Gould J,Koller D,Penn AA

更新日期：2010-09-08 00:00:00

abstract：:The epidermal growth factor receptor (EGFR) plays an important role in tissue homeostasis and tumor progression. However, cancer patients treated with EGFR inhibitors (EGFRIs) frequently develop acneiform skin toxicities, which are a strong predictor of a patient's treatment response. We show that the early inflammato...

journal_title：Science translational medicine

authors： Lichtenberger BM,Gerber PA,Holcmann M,Buhren BA,Amberg N,Smolle V,Schrumpf H,Boelke E,Ansari P,Mackenzie C,Wollenberg A,Kislat A,Fischer JW,Röck K,Harder J,Schröder JM,Homey B,Sibilia M

更新日期：2013-08-21 00:00:00

abstract：:Atherosclerotic lesional macrophages express molecules that promote plaque progression, but lack of mechanisms to therapeutically target these molecules represents a major gap in translational cardiovascular research. Here, we tested the efficacy of a small interfering RNA (siRNA) nanoparticle (NP) platform targeting ...

journal_title：Science translational medicine

authors： Tao W,Yurdagul A Jr,Kong N,Li W,Wang X,Doran AC,Feng C,Wang J,Islam MA,Farokhzad OC,Tabas I,Shi J

更新日期：2020-07-22 00:00:00

abstract：:Adding an electrical pulse to a microfluidic device that squeezes cells through narrow channels efficiently delivers DNA to the nucleus. ...

journal_title：Science translational medicine

authors： King KR

更新日期：2017-03-22 00:00:00

abstract：:Increased DNA breaks in aging skeletal muscle activate the DNA-PK pathway, whereas blocking this pathway improves mitochondrial density, physical fitness, body weight, and insulin resistance in mice. ...

journal_title：Science translational medicine

authors： Shoemaker AH

更新日期：2017-05-17 00:00:00

abstract：:Despite the high clinical burden, little is known about pathophysiology underlying autism spectrum disorder (ASD). Recent resting-state functional magnetic resonance imaging (rs-fMRI) studies have found atypical synchronization of brain activity in ASD. However, no consensus has been reached on the nature and clinical...

journal_title：Science translational medicine

authors： Holiga Š,Hipp JF,Chatham CH,Garces P,Spooren W,D'Ardhuy XL,Bertolino A,Bouquet C,Buitelaar JK,Bours C,Rausch A,Oldehinkel M,Bouvard M,Amestoy A,Caralp M,Gueguen S,Ly-Le Moal M,Houenou J,Beckmann CF,Loth E,Murphy D

更新日期：2019-02-27 00:00:00

abstract：:Hutchinson-Gilford progeria syndrome (HGPS) is caused by a point mutation in the LMNA gene that activates a cryptic donor splice site and yields a truncated form of prelamin A called progerin. Small amounts of progerin are also produced during normal aging. Studies with mouse models of HGPS have allowed the recent dev...

journal_title：Science translational medicine

authors： Osorio FG,Navarro CL,Cadiñanos J,López-Mejía IC,Quirós PM,Bartoli C,Rivera J,Tazi J,Guzmán G,Varela I,Depetris D,de Carlos F,Cobo J,Andrés V,De Sandre-Giovannoli A,Freije JM,Lévy N,López-Otín C

更新日期：2011-10-26 00:00:00

abstract：:Neuroinflammation has been increasingly recognized to play a critical role in Alzheimer's disease (AD). The epoxy fatty acids (EpFAs) are derivatives of the arachidonic acid metabolism pathway and have anti-inflammatory activities. However, their efficacy is limited because of their rapid hydrolysis by the soluble epo...

journal_title：Science translational medicine

authors： Ghosh A,Comerota MM,Wan D,Chen F,Propson NE,Hwang SH,Hammock BD,Zheng H

更新日期：2020-12-09 00:00:00