Surf's up for ER stress.

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：:Lapatinib, a dual HER2/EGFR (human epidermal growth factor receptor 2/epidermal growth factor receptor) inhibitor, is a recently approved targeted therapy for metastatic breast cancer. Because lapatinib enhances the efficacy of the chemotherapeutic agent capecitabine in breast cancer patients, we tested whether lapati...

journal_title：Science translational medicine

authors： Dolloff NG,Mayes PA,Hart LS,Dicker DT,Humphreys R,El-Deiry WS

更新日期：2011-06-08 00:00:00

abstract：:A congenital or iatrogenic tissue defect often requires closure by open surgery or metallic components that can erode tissue. Biodegradable, hydrophobic light-activated adhesives represent an attractive alternative to sutures, but lack a specifically designed minimally invasive delivery tool, which limits their clinic...

journal_title：Science translational medicine

authors： Roche ET,Fabozzo A,Lee Y,Polygerinos P,Friehs I,Schuster L,Whyte W,Casar Berazaluce AM,Bueno A,Lang N,Pereira MJ,Feins E,Wasserman S,O'Cearbhaill ED,Vasilyev NV,Mooney DJ,Karp JM,Del Nido PJ,Walsh CJ

更新日期：2015-09-23 00:00:00

abstract：:Tumor cells engineered to express therapeutic agents have shown promise to treat cancer. However, their potential to target cell surface receptors specific to the tumor site and their posttreatment fate have not been explored. We created therapeutic tumor cells expressing ligands specific to primary and recurrent tumo...

journal_title：Science translational medicine

authors： Reinshagen C,Bhere D,Choi SH,Hutten S,Nesterenko I,Wakimoto H,Le Roux E,Rizvi A,Du W,Minicucci C,Shah K

更新日期：2018-07-11 00:00:00

abstract：:Imaging, including its use for innovative tissue sampling, is slowly being recognized as playing a pivotal role in drug development, clinical trial design, and more effective delivery and monitoring of molecular therapies. The challenge is that, while a considerable number of new imaging technologies and new targeted ...

journal_title：Science translational medicine

authors： Weissleder R,Schwaiger MC,Gambhir SS,Hricak H

更新日期：2016-09-07 00:00:00

abstract：:Advances in understanding the genetics and underlying pathology of the catastrophic childhood epilepsies are pointing toward treatments. ...

journal_title：Science translational medicine

authors： Katsnelson A,Buzsáki G,Swann JW

更新日期：2014-11-12 00:00:00

abstract：:Diseases of failed inflammation resolution are common and largely incurable. Therapeutic induction of inflammation resolution is an attractive strategy to bring about healing without increasing susceptibility to infection. However, therapeutic targeting of inflammation resolution has been hampered by a lack of underst...

journal_title：Science translational medicine

authors： Robertson AL,Holmes GR,Bojarczuk AN,Burgon J,Loynes CA,Chimen M,Sawtell AK,Hamza B,Willson J,Walmsley SR,Anderson SR,Coles MC,Farrow SN,Solari R,Jones S,Prince LR,Irimia D,Rainger GE,Kadirkamanathan V,Whyte MK,Ren

更新日期：2014-02-26 00:00:00

abstract：:Myofibroblast-derived αvβ1 integrin has emerged as a critical mediator of transforming growth factor-β activation in experimental models of tissue fibrosis (Reed et al., this issue). ...

journal_title：Science translational medicine

authors： Dietz HC

更新日期：2015-05-20 00:00:00

abstract：:Direct-to-consumer (DTC) advertising of suspect goods and services has burgeoned because of the Internet. Despite very limited approval for use, DTC stem cell-marketed "treatments" have emerged for an array of conditions, creating global public health and safety risks. However, it remains unclear whether such use of s...

journal_title：Science translational medicine

authors： Liang BA,Mackey TK

更新日期：2012-09-12 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：: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：: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：:Research standards deviate in genetic versus nongenetic epidemiology. Besides some immutable differences, such as the correlation pattern between variables, these divergent research standards can converge considerably. Current research designs that dissociate genetic and nongenetic measurements are reaching their limi...

journal_title：Science translational medicine

authors： Ioannidis JP,Loy EY,Poulton R,Chia KS

更新日期：2009-11-18 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：:Acquired resistance to tyrosine kinase inhibitors (TKIs) of epidermal growth factor receptor (EGFR) remains a clinical challenge. Especially challenging are cases in which resistance emerges through EGFR-independent mechanisms, such as through pathways that promote epithelial-to-mesenchymal transition (EMT). Through a...

journal_title：Science translational medicine

authors： Nilsson MB,Sun H,Robichaux J,Pfeifer M,McDermott U,Travers J,Diao L,Xi Y,Tong P,Shen L,Hofstad M,Kawakami M,Le X,Liu X,Fan Y,Poteete A,Hu L,Negrao MV,Tran H,Dmitrovsky E,Peng D,Gibbons DL,Wang J,Heymach JV

更新日期：2020-09-02 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：:Inhaled drugs offer advantages, such as rapid onset of action, but require formulations and delivery systems that reproducibly and conveniently administer the drug. CVT-301 is a powder formulation of levodopa delivered by a breath-actuated inhaler that has been developed for treating OFF episodes (motor fluctuations b...

journal_title：Science translational medicine

authors： Lipp MM,Batycky R,Moore J,Leinonen M,Freed MI

更新日期：2016-10-12 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：: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：: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：:Merkel cell polyomavirus (MCV) causes ~80% of primary and metastatic Merkel cell carcinomas (MCCs). By comparing digital transcriptome subtraction deep-sequencing profiles, we found that transcripts of the cellular survivin oncoprotein [BIRC5a (baculoviral inhibitor of apoptosis repeat-containing 5)] were up-regulated...

journal_title：Science translational medicine

authors： Arora R,Shuda M,Guastafierro A,Feng H,Toptan T,Tolstov Y,Normolle D,Vollmer LL,Vogt A,Dömling A,Brodsky JL,Chang Y,Moore PS

更新日期：2012-05-09 00:00:00

abstract：:A primary barrier to the success of T cell-recruiting bispecific antibodies in the treatment of solid tumors is the lack of tumor-specific targets, resulting in on-target off-tumor adverse effects from T cell autoreactivity to target-expressing organs. To overcome this, we developed an anti-HER2/CD3 T cell-dependent b...

journal_title：Science translational medicine

authors： Slaga D,Ellerman D,Lombana TN,Vij R,Li J,Hristopoulos M,Clark R,Johnston J,Shelton A,Mai E,Gadkar K,Lo AA,Koerber JT,Totpal K,Prell R,Lee G,Spiess C,Junttila TT

更新日期：2018-10-17 00:00:00

abstract：:About 25% of high-grade cervical intraepithelial neoplasias (CIN2/3) caused by human papillomavirus serotype 16 (HPV16) undergo complete spontaneous regression. However, to date, therapeutic vaccination strategies for HPV disease have yielded limited success when measured by their ability to induce robust peripheral b...

journal_title：Science translational medicine

authors： Maldonado L,Teague JE,Morrow MP,Jotova I,Wu TC,Wang C,Desmarais C,Boyer JD,Tycko B,Robins HS,Clark RA,Trimble CL

更新日期：2014-01-29 00:00:00

abstract：:Current HIV vaccines are only partially efficacious, presenting an opportunity to identify correlates of protection and, thereby, potential insight into mechanisms that prevent HIV acquisition. Two independent preclinical challenge studies in nonhuman primates (NHPs) previously showed partial efficacy of a mosaic aden...

journal_title：Science translational medicine

authors： Ehrenberg PK,Shangguan S,Issac B,Alter G,Geretz A,Izumi T,Bryant C,Eller MA,Wegmann F,Apps R,Creegan M,Bolton DL,Sekaly RP,Robb ML,Gramzinski RA,Pau MG,Schuitemaker H,Barouch DH,Michael NL,Thomas R

更新日期：2019-08-28 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：:Much of our knowledge of the mechanisms underlying plasticity in the visual cortex in response to visual impairment, vision restoration, and environmental interactions comes from animal studies. We evaluated human brain plasticity in a group of patients with Leber's congenital amaurosis (LCA), who regained vision thro...

journal_title：Science translational medicine

authors： Ashtari M,Zhang H,Cook PA,Cyckowski LL,Shindler KS,Marshall KA,Aravand P,Vossough A,Gee JC,Maguire AM,Baker CI,Bennett J

更新日期：2015-07-15 00:00:00

abstract：:Public health emergencies, such as an Ebola disease outbreak, provide a complex and challenging environment for the evaluation of candidate vaccines. Here, we outline the need for flexible and responsive vaccine trial designs to be used in public health emergencies, and we summarize recommendations for their use in th...

journal_title：Science translational medicine

authors： Dean NE,Gsell PS,Brookmeyer R,De Gruttola V,Donnelly CA,Halloran ME,Jasseh M,Nason M,Riveros X,Watson CH,Henao-Restrepo AM,Longini IM

更新日期：2019-07-03 00:00:00

abstract：:Sustained viremia after acute HIV infection is associated with profound CD4+ T cell loss and exhaustion of HIV-specific CD8+ T cell responses. To determine the impact of combination antiretroviral therapy (cART) on these processes, we examined the evolution of immune responses in acutely infected individuals initiatin...

journal_title：Science translational medicine

authors： Ndhlovu ZM,Kazer SW,Nkosi T,Ogunshola F,Muema DM,Anmole G,Swann SA,Moodley A,Dong K,Reddy T,Brockman MA,Shalek AK,Ndung'u T,Walker BD

更新日期：2019-05-22 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：:Age is the most significant risk factor for atherosclerosis; however, the link between age and atherosclerosis is poorly understood. During both aging and atherosclerosis progression, the blood vessel wall stiffens owing to alterations in the extracellular matrix. Using in vitro and ex vivo models of vessel wall stiff...

journal_title：Science translational medicine

authors： Huynh J,Nishimura N,Rana K,Peloquin JM,Califano JP,Montague CR,King MR,Schaffer CB,Reinhart-King CA

更新日期：2011-12-07 00:00:00