siRNA nanoparticles targeting CaMKIIγ in lesional macrophages improve atherosclerotic plaque stability in mice.

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：: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：:Danon disease (DD) is a rare X-linked autophagic vacuolar myopathy associated with multiorgan dysfunction, including the heart, skeletal muscle, and liver. There are no specific treatments, and most male patients die from advanced heart failure during the second or third decade of life. DD is caused by mutations in th...

journal_title：Science translational medicine

authors： Manso AM,Hashem SI,Nelson BC,Gault E,Soto-Hermida A,Villarruel E,Brambatti M,Bogomolovas J,Bushway PJ,Chen C,Battiprolu P,Keravala A,Schwartz JD,Shah G,Gu Y,Dalton ND,Hammond K,Peterson K,Saftig P,Adler ED

更新日期：2020-03-18 00:00:00

abstract：:Recent studies highlighted long noncoding RNAs (lncRNAs) to play an important role in cardiac development. However, understanding of lncRNAs in cardiac diseases is still limited. Global lncRNA expression profiling indicated that several lncRNA transcripts are deregulated during pressure overload-induced cardiac hypert...

journal_title：Science translational medicine

authors： Viereck J,Kumarswamy R,Foinquinos A,Xiao K,Avramopoulos P,Kunz M,Dittrich M,Maetzig T,Zimmer K,Remke J,Just A,Fendrich J,Scherf K,Bolesani E,Schambach A,Weidemann F,Zweigerdt R,de Windt LJ,Engelhardt S,Dandekar T,

更新日期：2016-02-17 00:00:00

abstract：:We tested the hypothesis that ex vivo hepatocyte gene therapy can correct the metabolic disorder in fumarylacetoacetate hydrolase-deficient (Fah(-/-)) pigs, a large animal model of hereditary tyrosinemia type 1 (HT1). Recipient Fah(-/-) pigs underwent partial liver resection and hepatocyte isolation by collagenase dig...

journal_title：Science translational medicine

authors： Hickey RD,Mao SA,Glorioso J,Elgilani F,Amiot B,Chen H,Rinaldo P,Marler R,Jiang H,DeGrado TR,Suksanpaisan L,O'Connor MK,Freeman BL,Ibrahim SH,Peng KW,Harding CO,Ho CS,Grompe M,Ikeda Y,Lillegard JB,Russell SJ,Nybe

更新日期：2016-07-27 00:00:00

abstract：:About 170 million people worldwide are infected with hepatitis C virus (HCV). The current standard therapy leads to sustained viral elimination in only approximately 50% of the treated patients. Telaprevir, an HCV protease inhibitor, has substantial antiviral activity in patients with chronic HCV infection. However, i...

journal_title：Science translational medicine

authors： Rong L,Dahari H,Ribeiro RM,Perelson AS

更新日期：2010-05-05 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：:In 2013, chikungunya virus (CHIKV) transmission was documented in the Western Hemisphere, and the virus has since spread throughout the Americas with more than 1.8 million people infected in more than 40 countries. CHIKV targets the joints, resulting in symmetric polyarthritis that clinically mimics rheumatoid arthrit...

journal_title：Science translational medicine

authors： Miner JJ,Cook LE,Hong JP,Smith AM,Richner JM,Shimak RM,Young AR,Monte K,Poddar S,Crowe JE Jr,Lenschow DJ,Diamond MS

更新日期：2017-02-01 00:00:00

abstract：:Arrhythmogenic cardiomyopathy (ACM) is characterized by frequent cardiac arrhythmias. To elucidate the underlying mechanisms and discover potential chemical modifiers, we created a zebrafish model of ACM with cardiac myocyte-specific expression of the human 2057del2 mutation in the gene encoding plakoglobin. A high-th...

journal_title：Science translational medicine

authors： Asimaki A,Kapoor S,Plovie E,Karin Arndt A,Adams E,Liu Z,James CA,Judge DP,Calkins H,Churko J,Wu JC,MacRae CA,Kléber AG,Saffitz JE

更新日期：2014-06-11 00:00:00

abstract：:Currently, no vaccine exists for hepatitis C virus (HCV), a major pathogen thought to infect 170 million people globally. Many studies suggest that host T cell responses are critical for spontaneous resolution of disease, and preclinical studies have indicated a requirement for T cells in protection against challenge....

journal_title：Science translational medicine

authors： Barnes E,Folgori A,Capone S,Swadling L,Aston S,Kurioka A,Meyer J,Huddart R,Smith K,Townsend R,Brown A,Antrobus R,Ammendola V,Naddeo M,O'Hara G,Willberg C,Harrison A,Grazioli F,Esposito ML,Siani L,Traboni C,Oo Y

更新日期：2012-01-04 00:00:00

abstract：:Canavan disease is a hereditary leukodystrophy caused by mutations in the aspartoacylase gene (ASPA), leading to loss of enzyme activity and increased concentrations of the substrate N-acetyl-aspartate (NAA) in the brain. Accumulation of NAA results in spongiform degeneration of white matter and severe impairment of p...

journal_title：Science translational medicine

authors： Leone P,Shera D,McPhee SW,Francis JS,Kolodny EH,Bilaniuk LT,Wang DJ,Assadi M,Goldfarb O,Goldman HW,Freese A,Young D,During MJ,Samulski RJ,Janson CG

更新日期：2012-12-19 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：:In February 2010, the U.S. National Institutes of Health (NIH) sponsored a national Clinical and Translational Science Awards forum titled "Promoting Efficient and Effective Collaborations among Academia, Government and Industry." This forum brought together a broad set of stakeholders who were charged with developing...

journal_title：Science translational medicine

authors： Portilla LM,Evans G,Eng B,Fadem TJ

更新日期：2010-12-22 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：:Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease caused by loss of activity of α-l-iduronidase and attendant accumulation of the glycosaminoglycans dermatan sulfate and heparan sulfate. Current treatments are suboptimal and do not address residual disease including corneal clouding, skeletal deformi...

journal_title：Science translational medicine

authors： Dierenfeld AD,McEntee MF,Vogler CA,Vite CH,Chen AH,Passage M,Le S,Shah S,Jens JK,Snella EM,Kline KL,Parkes JD,Ware WA,Moran LE,Fales-Williams AJ,Wengert JA,Whitley RD,Betts DM,Boal AM,Riedesel EA,Gross W,Ellinwo

更新日期：2010-12-01 00:00:00

abstract：:Advances in health information technology and electronic medical records have the tremendous potential to accelerate translational and clinical research. However, privacy concerns threaten to be a rate-limiting factor. By recognizing and responding to patient privacy concerns, policy-makers, researchers, and informati...

journal_title：Science translational medicine

authors： Shelton RH

更新日期：2011-02-09 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：:Functionally diverse subpopulations of astrocytes have correlates in the neoplastic adult brain. ...

journal_title：Science translational medicine

authors： Venere M

更新日期：2017-03-01 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：:The limited predictability of phase II biomarkers for atherosclerosis outcomes in phase III studies stands in contrast to the number and varied types of biomarkers--soluble, imaging, and functional--that have been used in a diverse array of trials. Although collectively abundant, these biomarker data exist in a fragme...

journal_title：Science translational medicine

authors： Fryburg DA,Vassileva MT

更新日期：2011-03-02 00:00:00

abstract：:Osteoarthritis (OA) is a common degenerative condition that afflicts more than 70% of the population between 55 and 77 years of age. Although its prevalence is rising globally with aging of the population, current therapy is limited to symptomatic relief and, in severe cases, joint replacement surgery. We report that ...

journal_title：Science translational medicine

authors： Ruan MZ,Erez A,Guse K,Dawson B,Bertin T,Chen Y,Jiang MM,Yustein J,Gannon F,Lee BH

更新日期：2013-03-13 00:00:00

abstract：:Regulatory T cells (Tregs) are essential to prevent autoimmunity, but excessive Treg function contributes to cancer progression by inhibiting antitumor immune responses. Tregs exert contact-dependent inhibition of immune cells through the production of active transforming growth factor-β1 (TGF-β1). On the Treg cell su...

journal_title：Science translational medicine

authors： Cuende J,Liénart S,Dedobbeleer O,van der Woning B,De Boeck G,Stockis J,Huygens C,Colau D,Somja J,Delvenne P,Hannon M,Baron F,Dumoutier L,Renauld JC,De Haard H,Saunders M,Coulie PG,Lucas S

更新日期：2015-04-22 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：:Rapid advancements in the field of stem cell biology have led to many current efforts to exploit stem cells as therapeutic agents in regenerative medicine. However, current ex vivo cell manipulations common to most regenerative approaches create a variety of technical and regulatory hurdles to their clinical translati...

journal_title：Science translational medicine

authors： Arany PR,Cho A,Hunt TD,Sidhu G,Shin K,Hahm E,Huang GX,Weaver J,Chen AC,Padwa BL,Hamblin MR,Barcellos-Hoff MH,Kulkarni AB,J Mooney D

更新日期：2014-05-28 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：:Allergen-specific immunotherapy (AIT) has been used for more than 100 years as a tolerance-inducing therapy for allergic diseases and represents a potentially curative method of treatment. AIT functions through multiple mechanisms, including regulating T and B cell responses, changing antibody isotypes, and decreasing...

journal_title：Science translational medicine

authors： Akdis CA,Akdis M

更新日期：2015-03-25 00:00:00

abstract：:Antiretroviral therapy has improved the quality and length of life of millions of individuals affected by human immunodeficiency virus type 1 (HIV-1). The capacity of these drugs to indefinitely suppress HIV-which has a well-known capacity for escaping antiviral pressures-is surprising. In this issue of Science Transl...

journal_title：Science translational medicine

authors： Perelson AS,Deeks SG

更新日期：2011-07-13 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：: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：: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