Pitx2 modulates a Tbx5-dependent gene regulatory network to maintain atrial rhythm.

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：:Emerging viral diseases pose ongoing health threats, particularly in an era of globalization; however, new biomedical research technologies such as genome sequencing and structure-based vaccine and drug design have improved our ability to respond to viral threats. ...

journal_title：Science translational medicine

authors： Marston HD,Folkers GK,Morens DM,Fauci AS

更新日期：2014-09-10 00:00:00

abstract：:Despite the remarkable enhancements in breast cancer classification and characterization, an exhaustive comprehension of the underlying carcinogenic processes and an accurate prediction of its clinical behavior are yet to be achieved. On the wave of recent scientific advances and clinical findings, new cutting-edge te...

journal_title：Science translational medicine

authors： Reis-Filho JS,Weigelt B,Fumagalli D,Sotiriou C

更新日期：2010-09-01 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：: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：:Amyotrophic lateral sclerosis (ALS), a fatal disease causing progressive loss of motor neurons, still has no effective treatment. We developed a phenotypic screen to repurpose existing drugs using ALS motor neuron survival as readout. Motor neurons were generated from induced pluripotent stem cells (iPSCs) derived fro...

journal_title：Science translational medicine

authors： Imamura K,Izumi Y,Watanabe A,Tsukita K,Woltjen K,Yamamoto T,Hotta A,Kondo T,Kitaoka S,Ohta A,Tanaka A,Watanabe D,Morita M,Takuma H,Tamaoka A,Kunath T,Wray S,Furuya H,Era T,Makioka K,Okamoto K,Fujisawa T,Nishit

更新日期：2017-05-24 00:00:00

abstract：:Paving the way for clinical trials, a new study shows that a glutamate receptor antagonist reduces repetitive behavior and social deficits in a mouse model of autism spectrum disorder. ...

journal_title：Science translational medicine

authors： Gomez-Mancilla B

更新日期：2012-04-25 00:00:00

abstract：:Bactericidal antibiotics with diverse mechanisms of action induce generation of mitochondrial reactive oxygen species in mammalian cells (Kalghatgi et al., this issue). ...

journal_title：Science translational medicine

authors： Chandel NS,Budinger GR

更新日期：2013-07-03 00:00:00

abstract：:Retention and use, without explicit parental permission, of residual dried blood samples from newborn screening has generated public controversy over concerns about violations of family privacy rights and loss of parental autonomy. The public debate about this issue has included little discussion a...

journal_title：Science translational medicine

authors： Lewis MH,Scheurer ME,Green RC,McGuire AL

更新日期：2012-11-07 00:00:00

abstract：:Pathological angiogenesis is the hallmark of diseases such as cancer and retinopathies. Although tissue hypoxia and inflammation are recognized as central drivers of vessel growth, relatively little is known about the process that bridges the two. In a mouse model of ischemic retinopathy, we found that hypoxic regions...

journal_title：Science translational medicine

authors： Oubaha M,Miloudi K,Dejda A,Guber V,Mawambo G,Germain MA,Bourdel G,Popovic N,Rezende FA,Kaufman RJ,Mallette FA,Sapieha P

更新日期：2016-10-26 00:00:00

abstract：:Promising therapies for cartilage repair are translated through large animal models toward human application. To guide this work, regulatory agencies publish recommendations ("guidance documents") to direct pivotal large animal studies. These are meant to aid in study design, outline metrics for judging efficacy, and ...

journal_title：Science translational medicine

authors： Pfeifer CG,Fisher MB,Carey JL,Mauck RL

更新日期：2015-10-21 00:00:00

abstract：:Translation in an academic environment requires a support system--people, goals, models, partnerships, and infrastructures--that will push promising basic science and technology projects forward into the clinic. ...

journal_title：Science translational medicine

authors： Volk HD,Stevens MM,Mooney DJ,Grainger DW,Duda GN

更新日期：2015-04-08 00:00:00

abstract：:Nipah virus (NiV) is an emerging zoonotic paramyxovirus that causes severe and often fatal disease in pigs and humans. There are currently no vaccines or treatments approved for human use. Studies in small-animal models of NiV infection suggest that antibody therapy may be a promising treatment. However, most studies ...

journal_title：Science translational medicine

authors： Geisbert TW,Mire CE,Geisbert JB,Chan YP,Agans KN,Feldmann F,Fenton KA,Zhu Z,Dimitrov DS,Scott DP,Bossart KN,Feldmann H,Broder CC

更新日期：2014-06-25 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：: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：: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：:In recent years, there has been a growing focus on the unreliability of published biomedical and clinical research. To introduce effective new scientific contributors to the culture of health care, we propose a "datathon" or "hackathon" model in which participants with disparate, but potentially synergistic and comple...

journal_title：Science translational medicine

authors： Aboab J,Celi LA,Charlton P,Feng M,Ghassemi M,Marshall DC,Mayaud L,Naumann T,McCague N,Paik KE,Pollard TJ,Resche-Rigon M,Salciccioli JD,Stone DJ

更新日期：2016-04-06 00:00:00

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：:Aggregation of β-amyloid (Aβ) in the brain begins to occur years before the clinical onset of Alzheimer's disease (AD). Before Aβ aggregation, concentrations of extracellular soluble Aβ in the interstitial fluid (ISF) space of the brain, which are regulated by neuronal activity and the sleep-wake cycle, correlate with...

journal_title：Science translational medicine

authors： Roh JH,Huang Y,Bero AW,Kasten T,Stewart FR,Bateman RJ,Holtzman DM

更新日期：2012-09-05 00:00:00

abstract：:MicroRNAs (miRNAs) regulate many aspects of human biology. They target mRNAs for translational repression or degradation through base pairing with 3' untranslated regions, primarily via seed sequences (nucleotides 2 to 8 in the mature miRNA sequence). A number of individual miRNAs and miRNA families share seed sequenc...

journal_title：Science translational medicine

authors： Rottiers V,Obad S,Petri A,McGarrah R,Lindholm MW,Black JC,Sinha S,Goody RJ,Lawrence MS,deLemos AS,Hansen HF,Whittaker S,Henry S,Brookes R,Najafi-Shoushtari SH,Chung RT,Whetstine JR,Gerszten RE,Kauppinen S,Näär AM

更新日期：2013-11-20 00:00:00

abstract：:Aspirin use reduces the risk of colorectal neoplasia, at least in part, through inhibition of prostaglandin-endoperoxide synthase 2 (PTGS2, cyclooxygenase 2)-related pathways. Hydroxyprostaglandin dehydrogenase 15-(nicotinamide adenine dinucleotide) (15-PGDH, HPGD) is down-regulated in colorectal cancers and functions...

journal_title：Science translational medicine

authors： Fink SP,Yamauchi M,Nishihara R,Jung S,Kuchiba A,Wu K,Cho E,Giovannucci E,Fuchs CS,Ogino S,Markowitz SD,Chan AT

更新日期：2014-04-23 00:00:00

abstract：:Sleep disorders are highly prevalent in patients with traumatic brain injury (TBI) and can significantly impair cognitive rehabilitation. No proven therapies exist to mitigate the neurocognitive consequences of TBI. We show that mild brain injury in mice causes a persistent inability to maintain wakefulness and decrea...

journal_title：Science translational medicine

authors： Lim MM,Elkind J,Xiong G,Galante R,Zhu J,Zhang L,Lian J,Rodin J,Kuzma NN,Pack AI,Cohen AS

更新日期：2013-12-11 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：: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：: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：: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：: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 inability to produce perfusable microvasculature networks capable of supporting tissue survival and of withstanding physiological pressures without leakage is a fundamental problem facing the field of tissue engineering. Microvasculature is critically important for production of bioengineered lung (BEL), which req...

journal_title：Science translational medicine

authors： Nichols JE,La Francesca S,Niles JA,Vega SP,Argueta LB,Frank L,Christiani DC,Pyles RB,Himes BE,Zhang R,Li S,Sakamoto J,Rhudy J,Hendricks G,Begarani F,Liu X,Patrikeev I,Pal R,Usheva E,Vargas G,Miller A,Woodson L,

更新日期：2018-08-01 00:00:00

abstract：:Targeting androgen receptor (AR) axis signaling by disrupting androgen-AR interactions remains the primary treatment for metastatic prostate cancer. Unfortunately, all men develop resistance to primary castrating therapy and secondary androgen deprivation therapies (ADTs). Resistance develops in part because castratio...

journal_title：Science translational medicine

authors： Schweizer MT,Antonarakis ES,Wang H,Ajiboye AS,Spitz A,Cao H,Luo J,Haffner MC,Yegnasubramanian S,Carducci MA,Eisenberger MA,Isaacs JT,Denmeade SR

更新日期：2015-01-07 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