Vitamin C puts the pedal to the metal.

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：:Spinal cord injury (SCI) is typically complicated by progressive hemorrhagic necrosis, an autodestructive process of secondary injury characterized by progressive enlargement of a hemorrhagic contusion during the first several hours after trauma. We assessed the role of Abcc8, which encodes sulfonylurea receptor 1 (SU...

journal_title：Science translational medicine

authors： Simard JM,Woo SK,Norenberg MD,Tosun C,Chen Z,Ivanova S,Tsymbalyuk O,Bryan J,Landsman D,Gerzanich V

更新日期：2010-04-21 00:00:00

abstract：:Studies have suggested a role for the mammalian (or mechanistic) target of rapamycin (mTOR) in skeletal development and homeostasis, yet there is no evidence connecting mTOR with the key signaling pathways that regulate skeletogenesis. We identified a parathyroid hormone (PTH)/PTH-related peptide (PTHrP)-salt-inducibl...

journal_title：Science translational medicine

authors： Csukasi F,Duran I,Barad M,Barta T,Gudernova I,Trantirek L,Martin JH,Kuo CY,Woods J,Lee H,Cohn DH,Krejci P,Krakow D

更新日期：2018-09-19 00:00:00

abstract：:A preventive HIV-1 vaccine should induce HIV-1-specific broadly neutralizing antibodies (bnAbs). However, bnAbs generally require high levels of somatic hypermutation (SHM) to acquire breadth, and current vaccine strategies have not been successful in inducing bnAbs. Because bnAbs directed against a glycosylated site ...

journal_title：Science translational medicine

authors： Bonsignori M,Kreider EF,Fera D,Meyerhoff RR,Bradley T,Wiehe K,Alam SM,Aussedat B,Walkowicz WE,Hwang KK,Saunders KO,Zhang R,Gladden MA,Monroe A,Kumar A,Xia SM,Cooper M,Louder MK,McKee K,Bailer RT,Pier BW,Jette CA

更新日期：2017-03-15 00:00:00

abstract：:More than 100,000 genetic variants are reported to cause Mendelian disease in humans, but the penetrance-the probability that a carrier of the purported disease-causing genotype will indeed develop the disease-is generally unknown. We assess the impact of variants in the prion protein gene (PRNP) on the risk of prion ...

journal_title：Science translational medicine

authors： Minikel EV,Vallabh SM,Lek M,Estrada K,Samocha KE,Sathirapongsasuti JF,McLean CY,Tung JY,Yu LP,Gambetti P,Blevins J,Zhang S,Cohen Y,Chen W,Yamada M,Hamaguchi T,Sanjo N,Mizusawa H,Nakamura Y,Kitamoto T,Collins SJ,

更新日期：2016-01-20 00:00:00

abstract：:An oncolytic vaccinia virus expressing CXCL11 combined with PD-L1 blockade significantly reduces tumor burden and improves survival in murine cancer models. ...

journal_title：Science translational medicine

authors： Jonas BA

更新日期：2017-04-19 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 development of new approaches to cholera control relies on an accurate understanding of cholera epidemiology. However, most information on cholera incidence lacks laboratory confirmation and instead relies on surveillance systems reporting medically attended acute watery diarrhea. If recent infections could be ide...

journal_title：Science translational medicine

authors： Azman AS,Lessler J,Luquero FJ,Bhuiyan TR,Khan AI,Chowdhury F,Kabir A,Gurwith M,Weil AA,Harris JB,Calderwood SB,Ryan ET,Qadri F,Leung DT

更新日期：2019-02-20 00:00:00

abstract：:Clinical proteomics has yielded some early positive results-the identification of potential disease biomarkers-indicating the promise for this analytical approach to improve the current state of the art in clinical practice. However, the inability to verify some candidate molecules in subsequent studies has led to ske...

journal_title：Science translational medicine

authors： Mischak H,Allmaier G,Apweiler R,Attwood T,Baumann M,Benigni A,Bennett SE,Bischoff R,Bongcam-Rudloff E,Capasso G,Coon JJ,D'Haese P,Dominiczak AF,Dakna M,Dihazi H,Ehrich JH,Fernandez-Llama P,Fliser D,Frokiaer J,Garin

更新日期：2010-08-25 00:00:00

abstract：:Acute graft-versus-host disease (aGVHD) results from a robust response of donor T cells transferred during hematopoietic stem cell transplantation (HSCT) to allogeneic peptide-major histocompatibility complex antigens. Previous investigations have not identified T cell subsets that selectively mediate either protectiv...

journal_title：Science translational medicine

authors： Morris GP,Uy GL,Donermeyer D,Dipersio JF,Allen PM

更新日期：2013-06-05 00:00:00

abstract：:The August 2011 Clinical and Translational Science Awards conference "Using IT to Improve Community Health: How Health Care Reform Supports Innovation" convened four "Think Tank" sessions. Thirty individuals, representing various perspectives on community engagement, attended the "Health information technology (HIT) a...

journal_title：Science translational medicine

authors： Marriott LK,Nelson DA,Allen S,Calhoun K,Eldredge CE,Kimminau KS,Lucero RJ,Pineda-Reyes F,Rumala BB,Varanasi AP,Wasser JS,Shannon J

更新日期：2012-02-01 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：:Asthma is a clinically heterogeneous genetic disease, and its pathogenesis is incompletely understood. Genome-wide association studies link ORM (yeast)-Like protein isoform 3 [corrected] (ORMDL3), a member of the ORM gene family, to nonallergic childhood-onset asthma. Orm proteins negatively regulate sphingolipid (SL)...

journal_title：Science translational medicine

authors： Worgall TS,Veerappan A,Sung B,Kim BI,Weiner E,Bholah R,Silver RB,Jiang XC,Worgall S

更新日期：2013-05-22 00:00:00

abstract：:Genetic hearing loss accounts for up to 50% of prelingual deafness worldwide, yet there are no biologic treatments currently available. To investigate gene therapy as a potential biologic strategy for restoration of auditory function in patients with genetic hearing loss, we tested a gene augmentation approach in mous...

journal_title：Science translational medicine

authors： Askew C,Rochat C,Pan B,Asai Y,Ahmed H,Child E,Schneider BL,Aebischer P,Holt JR

更新日期：2015-07-08 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：:Touch perception on the fingers and hand is essential for fine motor control, contributes to our sense of self, allows for effective communication, and aids in our fundamental perception of the world. Despite increasingly sophisticated mechatronics, prosthetic devices still do not directly convey sensation back to the...

journal_title：Science translational medicine

authors： Tan DW,Schiefer MA,Keith MW,Anderson JR,Tyler J,Tyler DJ

更新日期：2014-10-08 00:00:00

abstract：:Each year, more than a half million people in the United States alone die from sepsis, a dire multisystem disease with highly inadequate treatment options. In a recent issue of Science, Puneet and colleagues provide compelling evidence that inhibiting sphingosine kinase 1--an enzyme that resides in immune cells and is...

journal_title：Science translational medicine

authors： O'Neill LA

更新日期：2010-06-16 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：: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：: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：: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：: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：: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：:The antiatherogenic drug nicotinic acid (niacin) has antidyslipidemic effects independent of free fatty acid suppression mediated by its receptor HCA₂ (GPR109A) (Lauring et al., this issue). ...

journal_title：Science translational medicine

authors： Offermanns S

更新日期：2012-08-22 00:00:00

abstract：:Macrophages in the healthy intestine are highly specialized and usually respond to the gut microbiota without provoking an inflammatory response. A breakdown in this tolerance leads to inflammatory bowel disease (IBD), but the mechanisms by which intestinal macrophages normally become conditioned to promote microbial ...

journal_title：Science translational medicine

authors： Scott NA,Andrusaite A,Andersen P,Lawson M,Alcon-Giner C,Leclaire C,Caim S,Le Gall G,Shaw T,Connolly JPR,Roe AJ,Wessel H,Bravo-Blas A,Thomson CA,Kästele V,Wang P,Peterson DA,Bancroft A,Li X,Grencis R,Mowat AM,Hal

更新日期：2018-10-24 00:00:00

abstract：:Cancer immunotherapy using antibodies that target immune checkpoints has delivered outstanding results. However, responses only occur in a subset of patients, and it is not fully understood what biological processes determine an effective outcome. This lack of understanding hinders the development of rational combinat...

journal_title：Science translational medicine

authors： Zemek RM,De Jong E,Chin WL,Schuster IS,Fear VS,Casey TH,Forbes C,Dart SJ,Leslie C,Zaitouny A,Small M,Boon L,Forrest ARR,Muiri DO,Degli-Esposti MA,Millward MJ,Nowak AK,Lassmann T,Bosco A,Lake RA,Lesterhuis WJ

更新日期：2019-07-17 00:00:00

abstract：:If discovery of new antibiotics continues to falter while resistance to drugs in clinical use continues to spread, society's medicine chest will soon lack effective treatments for many infections. Heritable antibiotic resistance emerges in bacteria from nonheritable resistance, also called phenotypic tolerance. This w...

journal_title：Science translational medicine

authors： Nathan C

更新日期：2012-06-27 00:00:00

abstract：:New therapeutic approaches are urgently needed to improve survival outcomes for patients with necrotizing pneumonia caused by Staphylococcus aureus One such approach is adjunctive treatment with intravenous immunoglobulin (IVIG), but clinical practice guidelines offer conflicting recommendations. In a preclinical rabb...

journal_title：Science translational medicine

authors： Diep BA,Le VT,Badiou C,Le HN,Pinheiro MG,Duong AH,Wang X,Dip EC,Aguiar-Alves F,Basuino L,Marbach H,Mai TT,Sarda MN,Kajikawa O,Matute-Bello G,Tkaczyk C,Rasigade JP,Sellman BR,Chambers HF,Lina G

更新日期：2016-09-21 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：:Alzheimer's disease (AD) is hypothesized to be caused by an overproduction or reduced clearance of amyloid-β (Aβ) peptide. Autosomal dominant AD (ADAD) caused by mutations in the presenilin (PSEN) gene have been postulated to result from increased production of Aβ42 compared to Aβ40 in the central nervous system (CNS)...

journal_title：Science translational medicine

authors： Potter R,Patterson BW,Elbert DL,Ovod V,Kasten T,Sigurdson W,Mawuenyega K,Blazey T,Goate A,Chott R,Yarasheski KE,Holtzman DM,Morris JC,Benzinger TL,Bateman RJ

更新日期：2013-06-12 00:00:00