Abstract:
:MG53 promotes sarcolemmal repair in the mdx mouse model of Duchenne muscular dystrophy (Weisleder et al., this issue) and identifies a new protein therapeutic for muscle disease.
journal_name
Sci Transl Medjournal_title
Science translational medicineauthors
Burkin DJ,Wuebbles RDdoi
10.1126/scitranslmed.3004082subject
Has Abstractpub_date
2012-06-20 00:00:00pages
139fs19issue
139eissn
1946-6234issn
1946-6242pii
4/139/139fs19journal_volume
4pub_type
评论,杂志文章abstract::Immune thrombocytopenia (ITP) is an autoimmune disorder of childhood characterized by immune-mediated destruction of platelets. The mechanisms underlying the pathogenesis of ITP and the therapeutic efficacy of intravenous immunoglobulins (IVIG) in this disorder remain unclear. We show that monocytes from patients with...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3006277
更新日期:2013-07-10 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
pub_type: 评论,杂志文章
doi:10.1126/scitranslmed.3010342
更新日期:2015-02-11 00:00:00
abstract::Most cancer deaths arise from metastases as a result of circulating tumor cells (CTCs) spreading from the primary tumor to vital organs. Despite progress in cancer prognosis, the role of CTCs in early disease diagnosis is unclear because of the low sensitivity of CTC assays. We demonstrate the high sensitivity of the ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aat5857
更新日期:2019-06-12 00:00:00
abstract::Cancer research integrated a physical sciences perspective through team science, which fostered communication, trust, joint publication, and open access to data. ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3005804
更新日期:2013-05-01 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3001380
更新日期:2010-12-01 00:00:00
abstract::We outline the fundamental properties of a highly participatory rapid learning system that can be developed in part from meaningful use of electronic health records (EHRs). Future widespread adoption of EHRs will make increasing amounts of medical information available in computable form. Secured and trusted use of th...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3001456
更新日期:2010-11-10 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
pub_type: 评论,杂志文章
doi:10.1126/scitranslmed.aab0044
更新日期:2015-05-20 00:00:00
abstract::Accumulating evidence has shown that repeated exposure to general anesthesia during critical stages of brain development results in long-lasting behavioral deficits later in life. To date, there has been no effective treatment to mitigate the neurotoxic effects of anesthesia on brain development. By performing calcium...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaf7151
更新日期:2016-06-22 00:00:00
abstract::Atherosclerotic lesional macrophages express molecules that promote plaque progression, but lack of mechanisms to therapeutically target these molecules represents a major gap in translational cardiovascular research. Here, we tested the efficacy of a small interfering RNA (siRNA) nanoparticle (NP) platform targeting ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aay1063
更新日期:2020-07-22 00:00:00
abstract::In severe spinal cord injuries, the tracts conveying motor commands to the spinal cord are disrupted, resulting in paralysis, but many patients still have small numbers of spared fibers. We have found that excitatory deep brain stimulation (DBS) of the mesencephalic locomotor region (MLR), an important control center ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3005972
更新日期:2013-10-23 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
pub_type: 临床试验,杂志文章
doi:10.1126/scitranslmed.aau0528
更新日期:2019-05-22 00:00:00
abstract::In acute and chronic inflammation, neutrophils and platelets, both of which promote monocyte recruitment, are often activated simultaneously. We investigated how secretory products of neutrophils and platelets synergize to enhance the recruitment of monocytes. We found that neutrophil-borne human neutrophil peptide 1 ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aad5330
更新日期:2015-12-09 00:00:00
abstract::Acquired ESR1 mutations are a major mechanism of resistance to aromatase inhibitors (AIs). We developed ultra high-sensitivity multiplex digital polymerase chain reaction assays for ESR1 mutations in circulating tumor DNA (ctDNA) and investigated the clinical relevance and origin of ESR1 mutations in 171 women with ad...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aac7551
更新日期:2015-11-11 00:00:00
abstract::Centronuclear myopathies (CNMs) are severe diseases characterized by muscle weakness and myofiber atrophy. Currently, there are no approved treatments for these disorders. Mutations in the phosphoinositide 3-phosphatase myotubularin (MTM1) are responsible for X-linked CNM (XLCNM), also called myotubular myopathy, wher...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aav1866
更新日期:2019-03-20 00:00:00
abstract::About one-third of people in the developed world will undergo cataract surgery in their lifetime. Although marked improvements in surgical technique have occurred since the development of the current approach to lens replacement in the late 1960s and early 1970s, some critical steps of the procedure can still only be ...
journal_title:Science translational medicine
pub_type: 临床试验,杂志文章
doi:10.1126/scitranslmed.3001305
更新日期:2010-11-17 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3002464
更新日期:2011-08-10 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.aba5146
更新日期:2020-07-08 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3001249
更新日期:2010-08-25 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
pub_type: 评论,杂志文章
doi:10.1126/scitranslmed.3004017
更新日期:2012-04-25 00:00:00
abstract::Long-acting GSK1265744 (GSK744 LA) is a strand transfer inhibitor of the HIV/SIV (simian immunodeficiency virus) integrase and was shown to be an effective preexposure prophylaxis (PrEP) agent in a low-dose intrarectal SHIV (simian-human immunodeficiency virus) rhesus macaque challenge model. We examined the pharmacok...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3010298
更新日期:2015-01-14 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
pub_type:
doi:10.1126/scitranslmed.3003363
更新日期:2012-02-01 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaf6984
更新日期:2017-12-20 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
pub_type:
doi:10.1126/scitranslmed.3010531
更新日期:2014-11-12 00:00:00
abstract::The Point-Person Project, a child-health research initiative, enables rapid response to opportunities for participation in multicenter pediatric clinical trials. ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaa2045
更新日期:2015-03-18 00:00:00
abstract::Numerous studies have implicated microRNAs (miRNAs) in cancer initiation and progression. In contrast, only recently has attention been focused on the pathway that generates these regulatory molecules. The identification of neoplasia-associated germline mutations in DICER1 has focused translational research on compone...
journal_title:Science translational medicine
pub_type: 杂志文章,评审
doi:10.1126/scitranslmed.3002493
更新日期:2011-11-30 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3002761
更新日期:2011-12-07 00:00:00
abstract::Patient-derived xenografts (PDXs) and PDX-derived cells (PDCs) are useful in preclinical research. We performed a drug screening assay using PDCs and identified proteasome inhibitors as promising drugs for cholangiocarcinoma (CCA) treatment. Furthermore, we determined that phosphate and tensin homology deleted on chro...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aay0152
更新日期:2020-09-23 00:00:00
abstract::Optogenetics offers promise for dissecting the complex neural circuits of the spinal cord and peripheral nervous system and has therapeutic potential for addressing unmet clinical needs. Much progress has been made to enable optogenetic control in normal and disease states, both in proof-of-concept and mechanistic stu...
journal_title:Science translational medicine
pub_type: 杂志文章,评审
doi:10.1126/scitranslmed.aad7577
更新日期:2016-05-04 00:00:00
abstract::Hepatitis B virus (HBV)-induced hepatitis and carcinogen-induced hepatocellular carcinoma (HCC) are associated with serum androgen concentration. However, how androgen or the androgen receptor (AR) contributes to HBV-induced hepatocarcinogenesis remains unclear. We found that hepatic AR promotes HBV-induced hepatocarc...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3001143
更新日期:2010-05-19 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3000124
更新日期:2009-10-07 00:00:00