A noncoding RNA antisense to moesin at 5p14.1 in autism.

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：:Variants in the leucine-rich repeat kinase-2 (LRRK2) gene are associated with Parkinson's disease, leprosy, and Crohn's disease, three disorders with inflammation as an important component. Because of its high expression in granulocytes and CD68-positive cells, LRRK2 may have a function in innate immunity. We tested t...

journal_title：Science translational medicine

authors： Shutinoski B,Hakimi M,Harmsen IE,Lunn M,Rocha J,Lengacher N,Zhou YY,Khan J,Nguyen A,Hake-Volling Q,El-Kodsi D,Li J,Alikashani A,Beauchamp C,Majithia J,Coombs K,Shimshek D,Marcogliese PC,Park DS,Rioux JD,Philpott D

更新日期：2019-09-25 00:00:00

abstract：:Retinal pigment epithelium (RPE) dysfunction and loss are a hallmark of non-neovascular age-related macular degeneration (NNAMD). Without the RPE, a majority of overlying photoreceptors ultimately degenerate, leading to severe, progressive vision loss. Clinical and histological studies suggest that RPE replacement str...

journal_title：Science translational medicine

authors： Kashani AH,Lebkowski JS,Rahhal FM,Avery RL,Salehi-Had H,Dang W,Lin CM,Mitra D,Zhu D,Thomas BB,Hikita ST,Pennington BO,Johnson LV,Clegg DO,Hinton DR,Humayun MS

更新日期：2018-04-04 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：: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：:In February 2010, the U.S. National Institutes of Health (NIH) sponsored a national Clinical and Translational Science Awards forum titled "Promoting Efficient and Effective Collaborations among Academia, Government and Industry." This forum brought together a broad set of stakeholders who were charged with developing...

journal_title：Science translational medicine

authors： Portilla LM,Evans G,Eng B,Fadem TJ

更新日期：2010-12-22 00:00:00

abstract：:With continued advances in science and technology, there is great potential to extend our healthspan as we age. ...

journal_title：Science translational medicine

authors： Dzau VJ,Finkelman EM,Balatbat CA,Verdin EM,Pettigrew RI

更新日期：2020-10-21 00:00:00

abstract：:Airway mucus in cystic fibrosis (CF) is highly elastic, but the mechanism behind this pathology is unclear. We hypothesized that the biophysical properties of CF mucus are altered because of neutrophilic oxidative stress. Using confocal imaging, rheology, and biochemical measures of inflammation and oxidation, we foun...

journal_title：Science translational medicine

authors： Yuan S,Hollinger M,Lachowicz-Scroggins ME,Kerr SC,Dunican EM,Daniel BM,Ghosh S,Erzurum SC,Willard B,Hazen SL,Huang X,Carrington SD,Oscarson S,Fahy JV

更新日期：2015-02-25 00:00:00

abstract：:Current HIV vaccines are only partially efficacious, presenting an opportunity to identify correlates of protection and, thereby, potential insight into mechanisms that prevent HIV acquisition. Two independent preclinical challenge studies in nonhuman primates (NHPs) previously showed partial efficacy of a mosaic aden...

journal_title：Science translational medicine

authors： Ehrenberg PK,Shangguan S,Issac B,Alter G,Geretz A,Izumi T,Bryant C,Eller MA,Wegmann F,Apps R,Creegan M,Bolton DL,Sekaly RP,Robb ML,Gramzinski RA,Pau MG,Schuitemaker H,Barouch DH,Michael NL,Thomas R

更新日期：2019-08-28 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：:The amyloid-forming proteins tau, αB crystallin, and amyloid P protein are all found in lesions of multiple sclerosis (MS). Our previous work established that amyloidogenic peptides from the small heat shock protein αB crystallin (HspB5) and from amyloid β fibrils, characteristic of Alzheimer's disease, were therapeut...

journal_title：Science translational medicine

authors： Kurnellas MP,Adams CM,Sobel RA,Steinman L,Rothbard JB

更新日期：2013-04-03 00:00:00

abstract：:Allergen-specific immunotherapy (AIT) has been used for more than 100 years as a tolerance-inducing therapy for allergic diseases and represents a potentially curative method of treatment. AIT functions through multiple mechanisms, including regulating T and B cell responses, changing antibody isotypes, and decreasing...

journal_title：Science translational medicine

authors： Akdis CA,Akdis M

更新日期：2015-03-25 00:00:00

abstract：:Acute tissue injury causes DNA damage and repair processes involving increased cell mitosis and polyploidization, leading to cell function alterations that may potentially drive cancer development. Here, we show that acute kidney injury (AKI) increased the risk for papillary renal cell carcinoma (pRCC) development and...

journal_title：Science translational medicine

authors： Peired AJ,Antonelli G,Angelotti ML,Allinovi M,Guzzi F,Sisti A,Semeraro R,Conte C,Mazzinghi B,Nardi S,Melica ME,De Chiara L,Lazzeri E,Lasagni L,Lottini T,Landini S,Giglio S,Mari A,Di Maida F,Antonelli A,Porpiglia F

更新日期：2020-03-25 00:00:00

abstract：:An innovative strategy for delivering drugs to chondrocytes in situ offers new avenues for treating osteoarthritis (Geiger et al, this issue). ...

journal_title：Science translational medicine

authors： Evans CH

更新日期：2018-11-28 00:00:00

abstract：:Antiretroviral therapy has improved the quality and length of life of millions of individuals affected by human immunodeficiency virus type 1 (HIV-1). The capacity of these drugs to indefinitely suppress HIV-which has a well-known capacity for escaping antiviral pressures-is surprising. In this issue of Science Transl...

journal_title：Science translational medicine

authors： Perelson AS,Deeks SG

更新日期：2011-07-13 00:00:00

abstract：:There is limited information about the impact of Zika virus (ZIKV) exposure in utero on the anti-ZIKV immune responses of offspring. We infected six rhesus macaque dams with ZIKV early or late in pregnancy and studied four of their offspring over the course of a year postpartum. Despite evidence of ZIKV exposure in ut...

journal_title：Science translational medicine

authors： Vannella KM,Stein S,Connelly M,Swerczek J,Amaro-Carambot E,Coyle EM,Babyak A,Winkler CW,Saturday G,Gai ND,Hammoud DA,Dowd KA,Valencia LP,Ramos-Benitez MJ,Kindrachuk J,Pierson TC,Peterson KE,Brenchley JM,Whitehead SS,

更新日期：2020-10-28 00:00:00

abstract：:The neurotransmitter glutamate is the primary excitatory neurotransmitter in mammalian brain and is responsible for most corticocortical and corticofugal neurotransmission. Disturbances in glutamatergic function have been implicated in the pathophysiology of several neuropsychiatric disorders-including schizophrenia, ...

journal_title：Science translational medicine

authors： Javitt DC,Schoepp D,Kalivas PW,Volkow ND,Zarate C,Merchant K,Bear MF,Umbricht D,Hajos M,Potter WZ,Lee CM

更新日期：2011-09-28 00:00:00

abstract：:Heparin-based anticoagulant drugs have been widely used for the prevention of blood clotting during surgical procedures and for the treatment of thromboembolic events. However, bleeding risks associated with these anticoagulants demand continuous monitoring and neutralization with suitable antidotes. Protamine, the on...

journal_title：Science translational medicine

authors： Shenoi RA,Kalathottukaren MT,Travers RJ,Lai BF,Creagh AL,Lange D,Yu K,Weinhart M,Chew BH,Du C,Brooks DE,Carter CJ,Morrissey JH,Haynes CA,Kizhakkedathu JN

更新日期：2014-10-29 00:00:00

abstract：:Physiologically relevant ALK (anaplastic lymphoma kinase) expression was not detected in human and mouse monocytes and macrophages, suggesting that the effects of bioactive compounds on stimulator of interferon genes (STING) activation may not depend on ALK. ...

journal_title：Science translational medicine

authors： Blasco RB,Patrucco E,Mota I,Tai WT,Chiarle R

更新日期：2018-12-12 00:00:00

abstract：:Epithelial tissues line the organs of the body, providing an initial protective barrier as well as a surface for nutrient and drug absorption. Here, we identified enzymatic components present in the gastrointestinal epithelium that can serve as selective means for tissue-directed polymerization. We focused on the smal...

journal_title：Science translational medicine

authors： Li J,Wang T,Kirtane AR,Shi Y,Jones A,Moussa Z,Lopes A,Collins J,Tamang SM,Hess K,Shakur R,Karandikar P,Lee JS,Huang HW,Hayward A,Traverso G

更新日期：2020-08-26 00:00:00

abstract：:Obesity promotes breast cancer by enhancing the stiffness of breast adipose tissue through changes in the extracellular matrix (Seo et al., this issue). ...

journal_title：Science translational medicine

authors： Arendt LM,Kuperwasser C

更新日期：2015-08-19 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：: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：:Adoptive cell transfer of ex vivo expanded regulatory T cells (Tregs) has shown immense potential in animal models of auto- and alloimmunity. However, the effective translation of such Treg therapies to the clinic has been slow. Because Treg homeostasis is known to require continuous T cell receptor (TCR) ligation and...

journal_title：Science translational medicine

authors： Eskandari SK,Sulkaj I,Melo MB,Li N,Allos H,Alhaddad JB,Kollar B,Borges TJ,Eskandari AS,Zinter MA,Cai S,Assaker JP,Choi JY,Al Dulaijan BS,Mansouri A,Haik Y,Tannous BA,van Son WJ,Leuvenink HGD,Pomahac B,Riella LV,

更新日期：2020-11-11 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：:High-grade B cell lymphomas with concurrent activation of the MYC and BCL2 oncogenes, also known as double-hit lymphomas (DHL), show dismal prognosis with current therapies. MYC activation sensitizes cells to inhibition of mitochondrial translation by the antibiotic tigecycline, and treatment with this compound provid...

journal_title：Science translational medicine

authors： Ravà M,D'Andrea A,Nicoli P,Gritti I,Donati G,Doni M,Giorgio M,Olivero D,Amati B

更新日期：2018-01-31 00:00:00

abstract：:Systemic lupus erythematosus (SLE) and discoid lupus erythematosus (DLE) of the skin are autoimmune diseases characterized by inappropriate immune responses against self-proteins; the key elements that determine disease pathogenesis and progression are largely unknown. Here, we show that mice lacking immune inhibitory...

journal_title：Science translational medicine

authors： Han X,Vesely MD,Yang W,Sanmamed MF,Badri T,Alawa J,López-Giráldez F,Gaule P,Lee SW,Zhang JP,Nie X,Nassar A,Boto A,Flies DB,Zheng L,Kim TK,Moeckel GW,McNiff JM,Chen L

更新日期：2019-12-11 00:00:00

abstract：:Case fatality rates for severe malaria remain high even in the best clinical settings because antimalarial drugs act against the parasite without alleviating life-threatening inflammation. We assessed the potential for host-directed therapy of severe malaria of a new class of anti-inflammatory drugs, the innate defens...

journal_title：Science translational medicine

authors： Achtman AH,Pilat S,Law CW,Lynn DJ,Janot L,Mayer ML,Ma S,Kindrachuk J,Finlay BB,Brinkman FS,Smyth GK,Hancock RE,Schofield L

更新日期：2012-05-23 00:00:00

abstract：:Cerebral cavernous malformation (CCM) is a genetic, cerebrovascular disease. Familial CCM is caused by genetic mutations in KRIT1, CCM2, or PDCD10 Disease onset is earlier and more severe in individuals with PDCD10 mutations. Recent studies have shown that lesions arise from excess mitogen-activated protein kinase kin...

journal_title：Science translational medicine

authors： Tang AT,Sullivan KR,Hong CC,Goddard LM,Mahadevan A,Ren A,Pardo H,Peiper A,Griffin E,Tanes C,Mattei LM,Yang J,Li L,Mericko-Ishizuka P,Shen L,Hobson N,Girard R,Lightle R,Moore T,Shenkar R,Polster SP,Roedel CJ,Li

更新日期：2019-11-27 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