Long-term follow-up after gene therapy for canavan disease.

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：:Beginning with the 18th-century physician-scientist William Heberden, the elder, Science Translational Medicine introduces a new article series about historical figures whose transformational contributions to science, medicine, and society remain relevant today. ...

journal_title：Science translational medicine

authors： Edelman ER,LaMarco K

更新日期：2015-05-13 00:00:00

abstract：:Pathogenic immunoglobulin G (IgG) autoantibodies characterize some human autoimmune diseases; their high concentration and long half-life are dependent on recycling by the neonatal Fc receptor (FcRn). Inhibition of FcRn is an attractive new treatment concept for IgG-mediated autoimmune diseases. Rozanolixizumab (UCB76...

journal_title：Science translational medicine

authors： Kiessling P,Lledo-Garcia R,Watanabe S,Langdon G,Tran D,Bari M,Christodoulou L,Jones E,Price G,Smith B,Brennan F,White I,Jolles S

更新日期：2017-11-01 00:00:00

abstract：:Treatment of life-threatening Epstein-Barr virus (EBV)-associated tumors remains a great challenge, especially for patients with relapsed or refractory disease. Here, we found that exosomes derived from phosphoantigen-expanded Vδ2-T cells (Vδ2-T-Exos) contained death-inducing ligands (FasL and TRAIL), an activating re...

journal_title：Science translational medicine

authors： Wang X,Xiang Z,Liu Y,Huang C,Pei Y,Wang X,Zhi H,Wong WH,Wei H,Ng IO,Lee PP,Chan GC,Lau YL,Tu W

更新日期：2020-09-30 00:00:00

abstract：:The research and development of innovative new therapies are expensive and risky processes, and companies require substantial resources if they are to translate basic science into modern biomedical products. When resources are limited, as in the current economic climate, biotechnology start-up companies are often amon...

journal_title：Science translational medicine

authors： Greenwood JC

更新日期：2010-01-06 00:00:00

abstract：:The Commentary of Mills and Sykes in Science Translational Medicine presented their thesis on the advent of high-throughput technologies and the dangers they may represent for the future of biomedical research. In response, we argue that true progress on the diagnosis and treatment of common human diseases will requir...

journal_title：Science translational medicine

authors： Lamb JR,Gibson NW

更新日期：2010-03-31 00:00:00

abstract：:The aim of treatment in congenital adrenal hyperplasia is to suppress excess adrenal androgens while achieving physiological glucocorticoid replacement. However, current glucocorticoid replacement regimes are inadequate because doses sufficient to suppress excess androgens almost invariably induce adverse metabolic ef...

journal_title：Science translational medicine

authors： Nixon M,Mackenzie SD,Taylor AI,Homer NZ,Livingstone DE,Mouras R,Morgan RA,Mole DJ,Stimson RH,Reynolds RM,Elfick AP,Andrew R,Walker BR

更新日期：2016-08-17 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：:Motor neuron-specific microRNA-218 (miR-218) has recently received attention because of its roles in mouse development. However, miR-218 relevance to human motor neuron disease was not yet explored. Here, we demonstrate by neuropathology that miR-218 is abundant in healthy human motor neurons. However, in amyotrophic ...

journal_title：Science translational medicine

authors： Reichenstein I,Eitan C,Diaz-Garcia S,Haim G,Magen I,Siany A,Hoye ML,Rivkin N,Olender T,Toth B,Ravid R,Mandelbaum AD,Yanowski E,Liang J,Rymer JK,Levy R,Beck G,Ainbinder E,Farhan SMK,Lennox KA,Bode NM,Behlke MA,

更新日期：2019-12-18 00:00:00

abstract：:Inherited retinal degenerative diseases, a genetically and phenotypically heterogeneous group of disorders, affect the function of photoreceptor cells and are among the leading causes of blindness. Recent advances in molecular genetics and cell biology are elucidating the pathophysiological mechanisms underlying these...

journal_title：Science translational medicine

authors： Scholl HP,Strauss RW,Singh MS,Dalkara D,Roska B,Picaud S,Sahel JA

更新日期：2016-12-07 00:00:00

abstract：:In multiple sclerosis (MS), lymphocyte--in particular B cell--transit between the central nervous system (CNS) and periphery may contribute to the maintenance of active disease. Clonally related B cells exist in the cerebrospinal fluid (CSF) and peripheral blood (PB) of MS patients; however, it remains unclear which s...

journal_title：Science translational medicine

authors： Palanichamy A,Apeltsin L,Kuo TC,Sirota M,Wang S,Pitts SJ,Sundar PD,Telman D,Zhao LZ,Derstine M,Abounasr A,Hauser SL,von Büdingen HC

更新日期：2014-08-06 00:00:00

abstract：:Attention deficit hyperactivity disorder (ADHD) is a common and persistent condition characterized by developmentally atypical and impairing inattention, hyperactivity, and impulsiveness. We identified de novo and rare copy number variations (CNVs) in 248 unrelated ADHD patients using million-feature genotyping arrays...

journal_title：Science translational medicine

authors： Lionel AC,Crosbie J,Barbosa N,Goodale T,Thiruvahindrapuram B,Rickaby J,Gazzellone M,Carson AR,Howe JL,Wang Z,Wei J,Stewart AF,Roberts R,McPherson R,Fiebig A,Franke A,Schreiber S,Zwaigenbaum L,Fernandez BA,Roberts W

更新日期：2011-08-10 00:00:00

abstract：:The mechanisms by which some HIV-infected subjects resist disease progression, whereas others progress rapidly, are incompletely understood. Viral and host genetic factors, such as nef deletions and major histocompatibility complex alleles, explain a portion of the observed variability. However, it has been difficult ...

journal_title：Science translational medicine

authors： Hartigan-O'Connor DJ,Abel K,Van Rompay KK,Kanwar B,McCune JM

更新日期：2012-05-30 00:00:00

abstract：:Disease-free infection in HIV-infected adults is associated with human leukocyte antigen-mediated suppression of viremia, whereas in the sooty mangabey and other healthy natural hosts of simian immunodeficiency virus (SIV), viral replication continues unabated. To better understand factors preventing HIV disease, we i...

journal_title：Science translational medicine

authors： Muenchhoff M,Adland E,Karimanzira O,Crowther C,Pace M,Csala A,Leitman E,Moonsamy A,McGregor C,Hurst J,Groll A,Mori M,Sinmyee S,Thobakgale C,Tudor-Williams G,Prendergast AJ,Kloverpris H,Roider J,Leslie A,Shingadia D

更新日期：2016-09-28 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：: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：:Functional magnetic resonance imaging offers an unprecedented opportunity to evaluate and compare drug effects on human brain activity and to provide systems-level predictions for how new drugs for chronic pain will affect the brain, thus accelerating drug discovery and repurposing (Duff et al., this issue). ...

journal_title：Science translational medicine

authors： Wager TD,Woo CW

更新日期：2015-02-11 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：: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：:Children who survive preterm birth exhibit persistent unexplained disturbances in cerebral cortical growth with associated cognitive and learning disabilities. The mechanisms underlying these deficits remain elusive. We used ex vivo diffusion magnetic resonance imaging to demonstrate in a preterm large-animal model th...

journal_title：Science translational medicine

authors： Dean JM,McClendon E,Hansen K,Azimi-Zonooz A,Chen K,Riddle A,Gong X,Sharifnia E,Hagen M,Ahmad T,Leigland LA,Hohimer AR,Kroenke CD,Back SA

更新日期：2013-01-16 00:00:00

abstract：:Raman spectroscopy is an optical imaging method that is based on the Raman effect, the inelastic scattering of a photon when energy is absorbed from light by a surface. Although Raman spectroscopy is widely used for chemical and molecular analysis, its clinical application has been hindered by the inherently weak natu...

journal_title：Science translational medicine

authors： Thakor AS,Luong R,Paulmurugan R,Lin FI,Kempen P,Zavaleta C,Chu P,Massoud TF,Sinclair R,Gambhir SS

更新日期：2011-04-20 00:00:00

abstract：:Immunization with the inactivated influenza vaccine (IIV) remains the most effective strategy to combat seasonal influenza infections. IIV activates B cells and T follicular helper (TFH) cells and thus engenders antibody-secreting cells and serum antibody titers. However, the cellular events preceding generation of pr...

journal_title：Science translational medicine

authors： Koutsakos M,Wheatley AK,Loh L,Clemens EB,Sant S,Nüssing S,Fox A,Chung AW,Laurie KL,Hurt AC,Rockman S,Lappas M,Loudovaris T,Mannering SI,Westall GP,Elliot M,Tangye SG,Wakim LM,Kent SJ,Nguyen THO,Kedzierska K

更新日期：2018-02-14 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：:Axonal dystrophy, indicative of perturbed axonal transport, occurs early during Alzheimer's disease (AD) pathogenesis. Little is known about the mechanisms underlying this initial sign of the pathology. This study proves that Golgi-localized γ-ear-containing ARF binding protein 3 (GGA3) loss of function, due to Gga3 g...

journal_title：Science translational medicine

authors： Lomoio S,Willen R,Kim W,Ho KZ,Robinson EK,Prokopenko D,Kennedy ME,Tanzi RE,Tesco G

更新日期：2020-11-18 00:00:00

abstract：:Platelets are crucial for normal hemostasis; however, their hyperactivation also contributes to many potentially lethal pathologies including myocardial infarction, stroke, and cancer. We hypothesized that modified platelets lacking their aggregation and activation capacity could act as reversible inhibitors of platel...

journal_title：Science translational medicine

authors： Papa AL,Jiang A,Korin N,Chen MB,Langan ET,Waterhouse A,Nash E,Caroff J,Graveline A,Vernet A,Mammoto A,Mammoto T,Jain A,Kamm RD,Gounis MJ,Ingber DE

更新日期：2019-02-13 00:00:00

abstract：:EphB receptors regulate the proliferation and positioning of intestinal stem and progenitor cells. In addition, they can act as tumor promoters for adenoma development but suppress progression to invasive carcinoma. We used imatinib to abrogate Abl kinase activity in Apc(Min/+) mice and in mice with LGR5(+) stem cells...

journal_title：Science translational medicine

authors： Kundu P,Genander M,Strååt K,Classon J,Ridgway RA,Tan EH,Björk J,Martling A,van Es J,Sansom OJ,Clevers H,Pettersson S,Frisén J

更新日期：2015-04-01 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：:Glycogen synthase kinase 3 (GSK3), a key regulatory kinase in the wingless-type MMTV integration site family (WNT) pathway, is a therapeutic target of interest in many diseases. Although dual GSK3α/β inhibitors have entered clinical trials, none has successfully translated to clinical application. Mechanism-based toxi...

journal_title：Science translational medicine

authors： Wagner FF,Benajiba L,Campbell AJ,Weïwer M,Sacher JR,Gale JP,Ross L,Puissant A,Alexe G,Conway A,Back M,Pikman Y,Galinsky I,DeAngelo DJ,Stone RM,Kaya T,Shi X,Robers MB,Machleidt T,Wilkinson J,Hermine O,Kung A,St

更新日期：2018-03-07 00:00:00

abstract：:Although children represent 25% of the U.S. population and nearly 40% of the world's population, pediatric product development typically lags behind that targeted at adults. Here the authors describe the challenges and opportunities of pediatric product development and the unique issues that surround pediatric researc...

journal_title：Science translational medicine

authors： Connor E,Lombardi D,van den Anker J

更新日期：2009-10-14 00:00:00

abstract：:Hutchinson-Gilford progeria syndrome (HGPS) is caused by a point mutation in the LMNA gene that activates a cryptic donor splice site and yields a truncated form of prelamin A called progerin. Small amounts of progerin are also produced during normal aging. Studies with mouse models of HGPS have allowed the recent dev...

journal_title：Science translational medicine

authors： Osorio FG,Navarro CL,Cadiñanos J,López-Mejía IC,Quirós PM,Bartoli C,Rivera J,Tazi J,Guzmán G,Varela I,Depetris D,de Carlos F,Cobo J,Andrés V,De Sandre-Giovannoli A,Freije JM,Lévy N,López-Otín C

更新日期：2011-10-26 00:00:00