Abstract:
:Cancer research integrated a physical sciences perspective through team science, which fostered communication, trust, joint publication, and open access to data.
journal_name
Sci Transl Medjournal_title
Science translational medicineauthors
Kuhn NZ,Nagahara LAdoi
10.1126/scitranslmed.3005804subject
Has Abstractpub_date
2013-05-01 00:00:00pages
183fs14, 1-3issue
183eissn
1946-6234issn
1946-6242pii
5/183/183fs14journal_volume
5pub_type
杂志文章abstract::Protein replacement therapies for rare monogenic diseases have a higher probability of regulatory approval compared with biologics, small molecules, and grant-funded orphan drugs. ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3005007
更新日期:2013-03-27 00:00:00
abstract::Cerebral cavernous malformation (CCM) is a genetic, cerebrovascular disease. Familial CCM is caused by genetic mutations in KRIT1, CCM2, or PDCD10 Disease onset is earlier and more severe in individuals with PDCD10 mutations. Recent studies have shown that lesions arise from excess mitogen-activated protein kinase kin...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaw3521
更新日期:2019-11-27 00:00:00
abstract::The U.S.-Latin America Cancer Research Network strives to expand translational research capabilities and forge new collaborations. ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aad5859
更新日期:2015-12-23 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::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::Obesity promotes breast cancer by enhancing the stiffness of breast adipose tissue through changes in the extracellular matrix (Seo et al., this issue). ...
journal_title:Science translational medicine
pub_type: 评论,杂志文章
doi:10.1126/scitranslmed.aac9446
更新日期:2015-08-19 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaa1983
更新日期:2015-04-22 00:00:00
abstract::Epithelial tissues line the organs of the body, providing an initial protective barrier as well as a surface for nutrient and drug absorption. Here, we identified enzymatic components present in the gastrointestinal epithelium that can serve as selective means for tissue-directed polymerization. We focused on the smal...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.abc0441
更新日期:2020-08-26 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::The brain is emerging as an important regulator of systemic glucose metabolism. Accumulating data from animal and observational human studies suggest that striatal dopamine signaling plays a role in glucose regulation, but direct evidence in humans is currently lacking. We present a series of experiments supporting th...
journal_title:Science translational medicine
pub_type: 杂志文章,随机对照试验
doi:10.1126/scitranslmed.aar3752
更新日期:2018-05-23 00:00:00
abstract::Rapid advances in the forward engineering of genetic circuitry in living cells has positioned synthetic biology as a potential means to solve numerous biomedical problems, including disease diagnosis and therapy. One challenge in exploiting synthetic biology for translational applications is to engineer microbes that ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaa3519
更新日期:2015-05-27 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::The epidermal growth factor receptor (EGFR) plays an important role in tissue homeostasis and tumor progression. However, cancer patients treated with EGFR inhibitors (EGFRIs) frequently develop acneiform skin toxicities, which are a strong predictor of a patient's treatment response. We show that the early inflammato...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3005886
更新日期:2013-08-21 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3000522
更新日期:2010-04-21 00:00:00
abstract::Alzheimer's disease (AD) is hypothesized to be caused by an overproduction or reduced clearance of amyloid-β (Aβ) peptide. Autosomal dominant AD (ADAD) caused by mutations in the presenilin (PSEN) gene have been postulated to result from increased production of Aβ42 compared to Aβ40 in the central nervous system (CNS)...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3005615
更新日期:2013-06-12 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaf1475
更新日期:2016-02-17 00:00:00
abstract::Improved understanding of the role of inflammation in tendon disease is required to facilitate therapeutic target discovery. We studied supraspinatus tendons from patients experiencing pain before and after surgical subacromial decompression treatment. Tendons were classified as having early, intermediate, or advanced...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aac4269
更新日期:2015-10-28 00:00:00
abstract::Sporadic inclusion body myositis (sIBM) is the commonest severe myopathy in patients more than 50 years of age. Previous therapeutic trials have targeted the inflammatory features of sIBM but all have failed. Because protein dyshomeostasis may also play a role in sIBM, we tested the effects of targeting this feature o...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aad4583
更新日期:2016-03-23 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.aab1996
更新日期:2015-07-08 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3006567
更新日期:2013-07-03 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.aaw3768
更新日期:2019-07-31 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.aag1048
更新日期:2016-09-28 00:00:00
abstract::A pilot study of a handheld fluorescence endomicroscope demonstrates both in vivo imaging and differentiation of normal from precancerous cervical tissue. ...
journal_title:Science translational medicine
pub_type: 评论,杂志文章
doi:10.1126/scitranslmed.aal4989
更新日期:2017-01-11 00:00:00
abstract::Overcoming drug resistance and targeting leukemic stem cells (LSCs) remain major challenges in curing BCR-ABL+ human leukemia. Using an advanced drug/proliferation screen, we have uncovered a prosurvival role for protein phosphatase 2A (PP2A) in tyrosine kinase inhibitor (TKI)-insensitive leukemic cells, regulated by ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aan8735
更新日期:2018-02-07 00:00:00
abstract::Acute graft-versus-host disease (aGVHD) results from a robust response of donor T cells transferred during hematopoietic stem cell transplantation (HSCT) to allogeneic peptide-major histocompatibility complex antigens. Previous investigations have not identified T cell subsets that selectively mediate either protectiv...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.3005452
更新日期:2013-06-05 00:00:00
abstract::Late infantile Batten disease (CLN2 disease) is an autosomal recessive, neurodegenerative lysosomal storage disease caused by mutations in the CLN2 gene encoding tripeptidyl peptidase 1 (TPP1). We tested intraparenchymal delivery of AAVrh.10hCLN2, a nonhuman serotype rh.10 adeno-associated virus vector encoding human ...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.abb5413
更新日期:2020-12-02 00:00:00
abstract::Chemotherapy-induced cardiotoxicity (CIC) is a common clinical problem that compromises effective anticancer therapies. Many chemotherapeutics (including anthracyclines, such as doxorubicin) induce the proapoptotic transcription factor p53 in the tumor and nonspecifically in the heart, promoting heart failure. Althoug...
journal_title:Science translational medicine
pub_type: 杂志文章
doi:10.1126/scitranslmed.aau8866
更新日期:2019-02-06 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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3007672
更新日期:2014-02-26 00:00:00
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::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
pub_type: 杂志文章
doi:10.1126/scitranslmed.3004291
更新日期:2012-09-05 00:00:00