Repurposed drug to the rescue of snakebite victims.

abstract：:Gene replacement and pre-mRNA splicing modifier therapies represent breakthrough gene targeting treatments for the neuromuscular disease spinal muscular atrophy (SMA), but mechanisms underlying variable efficacy of treatment are incompletely understood. Our examination of severe infantile onset human SMA tissues obtai...

journal_title：Science translational medicine

authors： Kong L,Valdivia DO,Simon CM,Hassinan CW,Delestrée N,Ramos DM,Park JH,Pilato CM,Xu X,Crowder M,Grzyb CC,King ZA,Petrillo M,Swoboda KJ,Davis C,Lutz CM,Stephan AH,Zhao X,Weetall M,Naryshkin NA,Crawford TO,Mentis GZ

更新日期：2021-01-27 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：: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：: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：: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：:There is a critical need for effective new pharmacotherapies for pain. The paucity of new drugs successfully reaching the clinic calls for a reassessment of current analgesic drug discovery approaches. Many points early in the discovery process present significant hurdles, making it critical to exploit advances in pai...

journal_title：Science translational medicine

authors： Borsook D,Hargreaves R,Bountra C,Porreca F

更新日期：2014-08-13 00:00:00

abstract：:Epidermal growth factor receptor (EGFR) gene mutations (G719X, exon 19 deletions/insertions, L858R, and L861Q) predict favorable responses to EGFR tyrosine kinase inhibitors (TKIs) in advanced non-small cell lung cancer (NSCLC). However, EGFR exon 20 insertion mutations (~10% of all EGFR mutations) are generally assoc...

journal_title：Science translational medicine

authors： Yasuda H,Park E,Yun CH,Sng NJ,Lucena-Araujo AR,Yeo WL,Huberman MS,Cohen DW,Nakayama S,Ishioka K,Yamaguchi N,Hanna M,Oxnard GR,Lathan CS,Moran T,Sequist LV,Chaft JE,Riely GJ,Arcila ME,Soo RA,Meyerson M,Eck MJ,K

更新日期：2013-12-18 00:00:00

abstract：:Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder. We screened 751 familial ALS patient whole-exome sequences and identified six mutations including p.D40G in the ANXA11 gene in 13 individuals. The p.D40G mutation was absent from 70,000 control whole-exome sequences. This mutation segregated wi...

journal_title：Science translational medicine

authors： Smith BN,Topp SD,Fallini C,Shibata H,Chen HJ,Troakes C,King A,Ticozzi N,Kenna KP,Soragia-Gkazi A,Miller JW,Sato A,Dias DM,Jeon M,Vance C,Wong CH,de Majo M,Kattuah W,Mitchell JC,Scotter EL,Parkin NW,Sapp PC,Nol

更新日期：2017-05-03 00:00:00

abstract：:Resveratrol is widely promoted as a potential cancer chemopreventive agent, but a lack of information on the optimal dose prohibits rationally designed trials to assess efficacy. To challenge the assumption that "more is better," we compared the pharmacokinetics and activity of a dietary dose with an intake 200 times ...

journal_title：Science translational medicine

authors： Cai H,Scott E,Kholghi A,Andreadi C,Rufini A,Karmokar A,Britton RG,Horner-Glister E,Greaves P,Jawad D,James M,Howells L,Ognibene T,Malfatti M,Goldring C,Kitteringham N,Walsh J,Viskaduraki M,West K,Miller A,Hemingwa

更新日期：2015-07-29 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：: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：:The tumor microenvironment is host to a complex network of cytokines that contribute to shaping the intratumoral immune reaction. Chromosomal gains and losses, coupled with expression analysis, of 59 cytokines and receptors and their functional networks were investigated in colorectal cancers. Changes in local express...

journal_title：Science translational medicine

authors： Mlecnik B,Bindea G,Angell HK,Sasso MS,Obenauf AC,Fredriksen T,Lafontaine L,Bilocq AM,Kirilovsky A,Tosolini M,Waldner M,Berger A,Fridman WH,Rafii A,Valge-Archer V,Pagès F,Speicher MR,Galon J

更新日期：2014-03-19 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：: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：:The AAA+ adenosine triphosphatase (ATPase) Thorase plays a critical role in controlling synaptic plasticity by regulating the expression of surface α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptors (AMPARs). Bidirectional sequencing of exons of ATAD1, the gene encoding Thorase, in a cohort of patients wit...

journal_title：Science translational medicine

authors： Umanah GKE,Pignatelli M,Yin X,Chen R,Crawford J,Neifert S,Scarffe L,Behensky AA,Guiberson N,Chang M,Ma E,Kim JW,Castro CC,Mao X,Chen L,Andrabi SA,Pletnikov MV,Pulver AE,Avramopoulos D,Bonci A,Valle D,Dawson TM,

更新日期：2017-12-13 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：:The elevation of carbon dioxide (CO2) in tissues and the bloodstream (hypercapnia) occurs in patients with severe lung diseases, including chronic obstructive pulmonary disease (COPD). Whereas hypercapnia has been recognized as a marker of COPD severity, a role for hypercapnia in disease pathogenesis remains unclear. ...

journal_title：Science translational medicine

authors： Shigemura M,Lecuona E,Angulo M,Homma T,Rodríguez DA,Gonzalez-Gonzalez FJ,Welch LC,Amarelle L,Kim SJ,Kaminski N,Budinger GRS,Solway J,Sznajder JI

更新日期：2018-09-05 00:00:00

abstract：:Local delivery of anticancer agents has the potential to maximize treatment efficacy and minimize the acute and long-term systemic toxicities. Here, we used unsupervised systematic evolution of ligands by exponential enrichment to identify four RNA aptamers that specifically recognized mouse and human myeloid cells in...

journal_title：Science translational medicine

authors： De La Fuente A,Zilio S,Caroli J,Van Simaeys D,Mazza EMC,Ince TA,Bronte V,Bicciato S,Weed DT,Serafini P

更新日期：2020-06-17 00:00:00

abstract：:Nitrogen-containing bisphosphonates (N-BPs), such as alendronate, are the most widely prescribed medications for diseases involving bone, with nearly 200 million prescriptions written annually. Recently, widespread use of N-BPs has been challenged due to the risk of rare but traumatic side effects such as atypical fem...

journal_title：Science translational medicine

authors： Surface LE,Burrow DT,Li J,Park J,Kumar S,Lyu C,Song N,Yu Z,Rajagopal A,Bae Y,Lee BH,Mumm S,Gu CC,Baker JC,Mohseni M,Sum M,Huskey M,Duan S,Bijanki VN,Civitelli R,Gardner MJ,McAndrew CM,Ricci WM,Gurnett CA,D

更新日期：2020-05-20 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：:Gene therapy is emerging as a therapeutic modality for treating disorders of the retina. Photoreceptor cells are the primary cell type affected in many inherited diseases of retinal degeneration. Successfully treating these diseases with gene therapy requires the identification of efficient and safe targeting vectors ...

journal_title：Science translational medicine

authors： Vandenberghe LH,Bell P,Maguire AM,Cearley CN,Xiao R,Calcedo R,Wang L,Castle MJ,Maguire AC,Grant R,Wolfe JH,Wilson JM,Bennett J

更新日期：2011-06-22 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：: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：:Prolonged life expectancy in the 20th century has been one of humankind's greatest triumphs. However, the substantial increase in the human life span has ushered in a new concern: healthy aging. Because infectious diseases prominently contribute to morbidity in the particularly vulnerable elderly population, strategie...

journal_title：Science translational medicine

authors： Boraschi D,Aguado MT,Dutel C,Goronzy J,Louis J,Grubeck-Loebenstein B,Rappuoli R,Del Giudice G

更新日期：2013-05-15 00:00:00

abstract：:The recent Perspective entitled "Power of rare diseases: Found in translation" undervalues the contributions of academic research in first-in-human studies. ...

journal_title：Science translational medicine

authors： van der Meer JW,Simon A,Dinarello CA

更新日期：2014-01-15 00:00:00

abstract：:Long synthetic peptides (LSPs) have a variety of important clinical uses as synthetic vaccines and drugs. Techniques for peptide synthesis were revolutionized in the 1960s and 1980s, after which efficient techniques for purification and characterization of the product were developed. These improved techniques allowed ...

journal_title：Science translational medicine

authors： Corradin G,Kajava AV,Verdini A

更新日期：2010-09-22 00:00:00

abstract：:Despite the existence of effective rabies vaccines for dogs, dog-transmitted human rabies persists and has reemerged in Africa. Two consecutive dog vaccination campaigns took place in Chad in 2012 and 2013 (coverage of 71% in both years) in the capital city of N'Djaména, as previously published. We developed a determi...

journal_title：Science translational medicine

authors： Zinsstag J,Lechenne M,Laager M,Mindekem R,Naïssengar S,Oussiguéré A,Bidjeh K,Rives G,Tessier J,Madjaninan S,Ouagal M,Moto DD,Alfaroukh IO,Muthiani Y,Traoré A,Hattendorf J,Lepelletier A,Kergoat L,Bourhy H,Dacheux L,

更新日期：2017-12-20 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：:The Wnt signaling pathway plays a central role in bone development and homeostasis. In most cases, Wnt ligands promote bone growth, which has led to speculation that Wnt factors could be used to stimulate bone healing. We gained insights into the mechanism by which Wnt signaling regulates adult bone repair through the...

journal_title：Science translational medicine

authors： Minear S,Leucht P,Jiang J,Liu B,Zeng A,Fuerer C,Nusse R,Helms JA

更新日期：2010-04-28 00:00:00

abstract：:PTEN (phosphatase and tensin homolog) loss of function is the most common genetic aberration in endometrioid endometrial carcinomas. In addition to its well-described role in cell signaling, PTEN is involved in the maintenance of genomic stability. Loss of PTEN function causes defects in repair of DNA double-strand br...

journal_title：Science translational medicine

authors： Dedes KJ,Wetterskog D,Mendes-Pereira AM,Natrajan R,Lambros MB,Geyer FC,Vatcheva R,Savage K,Mackay A,Lord CJ,Ashworth A,Reis-Filho JS

更新日期：2010-10-13 00:00:00