Stopping sepsis by targeting sphingosine kinase 1.

abstract：:Immunological and inflammatory processes downstream of dystrophin deficiency as well as metabolic abnormalities, defective autophagy, and loss of regenerative capacity all contribute to muscle pathology in Duchenne muscular dystrophy (DMD). These downstream cascades offer potential avenues for pharmacological interven...

journal_title：Science translational medicine

authors： Rosenberg AS,Puig M,Nagaraju K,Hoffman EP,Villalta SA,Rao VA,Wakefield LM,Woodcock J

更新日期：2015-08-05 00:00:00

abstract：:Acute myeloid leukemia (AML) is a molecularly and clinically heterogeneous hematological malignancy. Although immunotherapy may be an attractive modality to exploit in patients with AML, the ability to predict the groups of patients and the types of cancer that will respond to immune targeting remains limited. This st...

journal_title：Science translational medicine

authors： Vadakekolathu J,Minden MD,Hood T,Church SE,Reeder S,Altmann H,Sullivan AH,Viboch EJ,Patel T,Ibrahimova N,Warren SE,Arruda A,Liang Y,Smith TH,Foulds GA,Bailey MD,Gowen-MacDonald J,Muth J,Schmitz M,Cesano A,Pockley

更新日期：2020-06-03 00:00:00

abstract：:A congenital or iatrogenic tissue defect often requires closure by open surgery or metallic components that can erode tissue. Biodegradable, hydrophobic light-activated adhesives represent an attractive alternative to sutures, but lack a specifically designed minimally invasive delivery tool, which limits their clinic...

journal_title：Science translational medicine

authors： Roche ET,Fabozzo A,Lee Y,Polygerinos P,Friehs I,Schuster L,Whyte W,Casar Berazaluce AM,Bueno A,Lang N,Pereira MJ,Feins E,Wasserman S,O'Cearbhaill ED,Vasilyev NV,Mooney DJ,Karp JM,Del Nido PJ,Walsh CJ

更新日期：2015-09-23 00:00:00

abstract：:Information provided to patients is thought to influence placebo and drug effects. In a prospective, within-subjects, repeated-measures study of 66 subjects with episodic migraine, we investigated how variations in medication labeling modified placebo and drug effects. An initial attack with no treatment served as a c...

journal_title：Science translational medicine

authors： Kam-Hansen S,Jakubowski M,Kelley JM,Kirsch I,Hoaglin DC,Kaptchuk TJ,Burstein R

更新日期：2014-01-08 00:00:00

abstract：:Metastasis is the major cause of cancer mortality. A more thorough understanding of the mechanisms driving this complex multistep process will aid in the identification and characterization of therapeutically targetable genetic drivers of disease progression. We demonstrate that KLF6-SV1, an oncogenic splice variant o...

journal_title：Science translational medicine

authors： Hatami R,Sieuwerts AM,Izadmehr S,Yao Z,Qiao RF,Papa L,Look MP,Smid M,Ohlssen J,Levine AC,Germain D,Burstein D,Kirschenbaum A,DiFeo A,Foekens JA,Narla G

更新日期：2013-01-23 00:00:00

abstract：:Centronuclear myopathies (CNMs) are severe diseases characterized by muscle weakness and myofiber atrophy. Currently, there are no approved treatments for these disorders. Mutations in the phosphoinositide 3-phosphatase myotubularin (MTM1) are responsible for X-linked CNM (XLCNM), also called myotubular myopathy, wher...

journal_title：Science translational medicine

authors： Lionello VM,Nicot AS,Sartori M,Kretz C,Kessler P,Buono S,Djerroud S,Messaddeq N,Koebel P,Prokic I,Hérault Y,Romero NB,Laporte J,Cowling BS

更新日期：2019-03-20 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：: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：: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：: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：:Buprenorphine, a synthetic opioid that acts at both μ and κ opioid receptors, can decrease cocaine use in individuals with opioid addiction. However, the potent agonist action of buprenorphine at μ opioid receptors raises its potential for creating opioid dependence in non-opioid-dependent cocaine abusers. Here, we te...

journal_title：Science translational medicine

authors： Wee S,Vendruscolo LF,Misra KK,Schlosburg JE,Koob GF

更新日期：2012-08-08 00:00:00

abstract：:Previous attempts to gain insight into the pathogenesis of psoriasis and eczema by comparing their molecular signatures were hampered by the high interindividual variability of those complex diseases. In patients affected by both psoriasis and nonatopic or atopic eczema simultaneously (n = 24), an intraindividual comp...

journal_title：Science translational medicine

authors： Quaranta M,Knapp B,Garzorz N,Mattii M,Pullabhatla V,Pennino D,Andres C,Traidl-Hoffmann C,Cavani A,Theis FJ,Ring J,Schmidt-Weber CB,Eyerich S,Eyerich K

更新日期：2014-07-09 00:00:00

abstract：:Large bone defects cannot form a callus and exhibit high complication rates even with the best treatment strategies available. Tissue engineering approaches often use scaffolds designed to match the properties of mature bone. However, natural fracture healing is most efficient when it recapitulates development, formin...

journal_title：Science translational medicine

authors： McDermott AM,Herberg S,Mason DE,Collins JM,Pearson HB,Dawahare JH,Tang R,Patwa AN,Grinstaff MW,Kelly DJ,Alsberg E,Boerckel JD

更新日期：2019-06-05 00:00:00

abstract：:Fibroblast growth factor 21 (FGF21) is a distinctive member of the FGF family with potent beneficial effects on lipid, body weight, and glucose metabolism and has attracted considerable interest as a potential therapeutic for treating diabetes and obesity. As an alternative to native FGF21, we have developed a monoclo...

journal_title：Science translational medicine

authors： Foltz IN,Hu S,King C,Wu X,Yang C,Wang W,Weiszmann J,Stevens J,Chen JS,Nuanmanee N,Gupte J,Komorowski R,Sekirov L,Hager T,Arora T,Ge H,Baribault H,Wang F,Sheng J,Karow M,Wang M,Luo Y,McKeehan W,Wang Z,Vénia

更新日期：2012-11-28 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：:Migraine-related photophobia is multifaceted and may be mediated by numerous pathways. ...

journal_title：Science translational medicine

authors： Lehtinen MK

更新日期：2017-07-12 00:00:00

abstract：:Vaccines directed against the blood stages of Plasmodium falciparum malaria are intended to prevent the parasite from invading and replicating within host cells. No blood-stage malaria vaccine has shown clinical efficacy in humans. Most malaria vaccine antigens are parasite surface proteins that have evolved extensive...

journal_title：Science translational medicine

authors： Takala SL,Coulibaly D,Thera MA,Batchelor AH,Cummings MP,Escalante AA,Ouattara A,Traoré K,Niangaly A,Djimdé AA,Doumbo OK,Plowe CV

更新日期：2009-10-14 00:00:00

abstract：:Rapid antimicrobial susceptibility testing (AST) is urgently needed for informing treatment decisions and preventing the spread of antimicrobial resistance resulting from the misuse and overuse of antibiotics. To date, no phenotypic AST exists that can be performed within a single patient visit (30 min) directly from ...

journal_title：Science translational medicine

authors： Schoepp NG,Schlappi TS,Curtis MS,Butkovich SS,Miller S,Humphries RM,Ismagilov RF

更新日期：2017-10-04 00:00:00

abstract：:Next-generation sequencing technologies have greatly expanded our understanding of cancer genetics. Antisense technology is an attractive platform with the potential to translate these advances into improved cancer therapeutics, because antisense oligonucleotide (ASO) inhibitors can be designed on the basis of gene se...

journal_title：Science translational medicine

authors： Hong D,Kurzrock R,Kim Y,Woessner R,Younes A,Nemunaitis J,Fowler N,Zhou T,Schmidt J,Jo M,Lee SJ,Yamashita M,Hughes SG,Fayad L,Piha-Paul S,Nadella MV,Mohseni M,Lawson D,Reimer C,Blakey DC,Xiao X,Hsu J,Revenko A

更新日期：2015-11-18 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：: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：:Clinical advancement of the bioartificial liver is hampered by the lack of expandable human hepatocytes and appropriate bioreactors and carriers to encourage hepatic cells to function during extracorporeal circulation. We have recently developed an efficient approach for derivation of expandable liver progenitor-like ...

journal_title：Science translational medicine

authors： Li WJ,Zhu XJ,Yuan TJ,Wang ZY,Bian ZQ,Jing HS,Shi X,Chen CY,Fu GB,Huang WJ,Shi YP,Liu Q,Zeng M,Zhang HD,Wu HP,Yu WF,Zhai B,Yan HX

更新日期：2020-07-08 00:00:00

abstract：:Sporadic inclusion body myositis (sIBM) is the commonest severe myopathy in patients more than 50 years of age. Previous therapeutic trials have targeted the inflammatory features of sIBM but all have failed. Because protein dyshomeostasis may also play a role in sIBM, we tested the effects of targeting this feature o...

journal_title：Science translational medicine

authors： Ahmed M,Machado PM,Miller A,Spicer C,Herbelin L,He J,Noel J,Wang Y,McVey AL,Pasnoor M,Gallagher P,Statland J,Lu CH,Kalmar B,Brady S,Sethi H,Samandouras G,Parton M,Holton JL,Weston A,Collinson L,Taylor JP,Schia

更新日期：2016-03-23 00:00:00

abstract：:Pregnancy imposes a substantial metabolic burden on women through weight gain and insulin resistance. Lactation reduces the risk of maternal postpartum diabetes, but the mechanisms underlying this benefit are unknown. Here, we identified long-term beneficial effects of lactation on β cell function, which last for year...

journal_title：Science translational medicine

authors： Moon JH,Kim H,Kim H,Park J,Choi W,Choi W,Hong HJ,Ro HJ,Jun S,Choi SH,Banerjee RR,Shong M,Cho NH,Kim SK,German MS,Jang HC,Kim H

更新日期：2020-04-29 00:00:00

abstract：:Age is the most significant risk factor for atherosclerosis; however, the link between age and atherosclerosis is poorly understood. During both aging and atherosclerosis progression, the blood vessel wall stiffens owing to alterations in the extracellular matrix. Using in vitro and ex vivo models of vessel wall stiff...

journal_title：Science translational medicine

authors： Huynh J,Nishimura N,Rana K,Peloquin JM,Califano JP,Montague CR,King MR,Schaffer CB,Reinhart-King CA

更新日期：2011-12-07 00:00:00

abstract：:Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) anion channel. The most common CF-associated mutation is ΔF508, which deletes a phenylalanine in position 508. In vitro studies indicate that the resultant prot...

journal_title：Science translational medicine

authors： Ostedgaard LS,Meyerholz DK,Chen JH,Pezzulo AA,Karp PH,Rokhlina T,Ernst SE,Hanfland RA,Reznikov LR,Ludwig PS,Rogan MP,Davis GJ,Dohrn CL,Wohlford-Lenane C,Taft PJ,Rector MV,Hornick E,Nassar BS,Samuel M,Zhang Y,Richt

更新日期：2011-03-16 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：:As observed during the 2013-2016 Ebola virus disease epidemic, containment of filovirus outbreaks is challenging and made more difficult by the lack of approved vaccine or therapeutic options. Marburg and Ravn viruses are highly virulent and cause severe and frequently lethal disease in humans. Monoclonal antibodies (...

journal_title：Science translational medicine

authors： Mire CE,Geisbert JB,Borisevich V,Fenton KA,Agans KN,Flyak AI,Deer DJ,Steinkellner H,Bohorov O,Bohorova N,Goodman C,Hiatt A,Kim DH,Pauly MH,Velasco J,Whaley KJ,Crowe JE Jr,Zeitlin L,Geisbert TW

更新日期：2017-04-05 00:00:00

abstract：:Thanks to the revolutions in information technology, human "-omics" research, and intricate medical device development, academic health centers (AHCs) now have an unparalleled potential to become translational engines that both generate basic science advances and then translate them into human studies and, ultimately,...

journal_title：Science translational medicine

authors： Crowley WF Jr,Gusella JF

更新日期：2009-10-07 00:00:00

abstract：:Conventional radiation therapy for cancer usually consists of multiple treatments (called fractions) with low doses of radiation. These dose schemes are planned with the guidance of the linear-quadratic (LQ) model, which has been the most prevalent model for designing dose schemes in radiation therapy. The high-dose f...

journal_title：Science translational medicine

authors： Wang JZ,Huang Z,Lo SS,Yuh WT,Mayr NA

更新日期：2010-07-07 00:00:00