Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1.

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

authors： Saleme B,Gurtu V,Zhang Y,Kinnaird A,Boukouris AE,Gopal K,Ussher JR,Sutendra G

更新日期：2019-02-06 00:00:00

abstract：:The efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) is limited by acute and chronic graft-versus-host disease (GVHD). The impact of obesity on allo-HSCT outcomes is poorly understood. Here, we report that obesity had a negative and selective impact on acute gut GVHD after allo-HSCT in mice w...

journal_title：Science translational medicine

authors： Khuat LT,Le CT,Pai CS,Shields-Cutler RR,Holtan SG,Rashidi A,Parker SL,Knights D,Luna JI,Dunai C,Wang Z,Sturgill IR,Stoffel KM,Merleev AA,More SK,Maverakis E,Raybould HE,Chen M,Canter RJ,Monjazeb AM,Dave M,Ferrar

更新日期：2020-11-25 00:00:00

abstract：:Clinical management of human cancer is dependent on the accurate monitoring of residual and recurrent tumors. The evaluation of patient-specific translocations in leukemias and lymphomas has revolutionized diagnostics for these diseases. We have developed a method, called personalized analysis of rearranged ends (PARE...

journal_title：Science translational medicine

authors： Leary RJ,Kinde I,Diehl F,Schmidt K,Clouser C,Duncan C,Antipova A,Lee C,McKernan K,De La Vega FM,Kinzler KW,Vogelstein B,Diaz LA Jr,Velculescu VE

更新日期：2010-02-24 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

authors： Kuhn NZ,Nagahara LA

更新日期：2013-05-01 00:00:00

abstract：:Large bone defects cannot form a callus and exhibit high complication rates even with the best treatment strategies available. Tissue engineering approaches often use scaffolds designed to match the properties of mature bone. However, natural fracture healing is most efficient when it recapitulates development, formin...

journal_title：Science translational medicine

authors： McDermott AM,Herberg S,Mason DE,Collins JM,Pearson HB,Dawahare JH,Tang R,Patwa AN,Grinstaff MW,Kelly DJ,Alsberg E,Boerckel JD

更新日期：2019-06-05 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：:With continued advances in science and technology, there is great potential to extend our healthspan as we age. ...

journal_title：Science translational medicine

authors： Dzau VJ,Finkelman EM,Balatbat CA,Verdin EM,Pettigrew RI

更新日期：2020-10-21 00:00:00

abstract：:A major obstacle to eradicating polio is that poliovirus from endemic countries can be reintroduced to polio-free countries. Environmental surveillance (ES) can detect poliovirus from sewage or wastewaters samples, even in the absence of patients with paralysis. ES is underused, in part because its sensitivity is unkn...

journal_title：Science translational medicine

authors： Berchenko Y,Manor Y,Freedman LS,Kaliner E,Grotto I,Mendelson E,Huppert A

更新日期：2017-03-29 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

authors： Davis JM,Smoyer WE,Connor EM,Huskins WC,Pastern C,Purucker M,Schrader EC,Jackson CR,Kaskel FJ,Hirschfeld S

更新日期：2015-03-18 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

authors： Ahmed M,Machado PM,Miller A,Spicer C,Herbelin L,He J,Noel J,Wang Y,McVey AL,Pasnoor M,Gallagher P,Statland J,Lu CH,Kalmar B,Brady S,Sethi H,Samandouras G,Parton M,Holton JL,Weston A,Collinson L,Taylor JP,Schia

更新日期：2016-03-23 00:00:00

abstract：:Global investments in health information technology (HIT) in the form of electronic health record systems represent important new strategies to improve the quality and efficiency of health care. These potential benefits of HIT will not be achieved without direct attention to the use of health care information for clin...

journal_title：Science translational medicine

authors： Nadler JJ,Downing GJ

更新日期：2010-02-10 00:00:00

abstract：:Meibomian glands (MGs) are sebaceous glands of the eyelid margin that secrete lipids needed to avert tear evaporation and to help maintain ocular surface homeostasis. Obstruction of MGs or other forms of MG dysfunction can promote chronic diseases of the ocular surface. Although chronic eyelid inflammation, such as al...

journal_title：Science translational medicine

authors： Reyes NJ,Yu C,Mathew R,Kunnen CM,Kalnitsky J,Redfern RL,Leonardi A,Perez VL,MacLeod AS,Gupta PK,Saban DR

更新日期：2018-07-25 00:00:00

abstract：:Pluripotent stem cells are a scalable source of pancreatic cells for transplantation into patients with diabetes. Here, we describe how the field is gaining momentum toward a β cell replacement therapy. ...

journal_title：Science translational medicine

authors： Bruin JE,Rezania A,Kieffer TJ

更新日期：2015-12-02 00:00:00

abstract：:A preventive HIV-1 vaccine should induce HIV-1-specific broadly neutralizing antibodies (bnAbs). However, bnAbs generally require high levels of somatic hypermutation (SHM) to acquire breadth, and current vaccine strategies have not been successful in inducing bnAbs. Because bnAbs directed against a glycosylated site ...

journal_title：Science translational medicine

authors： Bonsignori M,Kreider EF,Fera D,Meyerhoff RR,Bradley T,Wiehe K,Alam SM,Aussedat B,Walkowicz WE,Hwang KK,Saunders KO,Zhang R,Gladden MA,Monroe A,Kumar A,Xia SM,Cooper M,Louder MK,McKee K,Bailer RT,Pier BW,Jette CA

更新日期：2017-03-15 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：:Cooperative oncogenic effects resulting from the loss of PTEN and overexpression of MYC overcome the deleterious effects of endoplasmic reticulum stress not only to promote the growth of aggressive prostate cancer but also to expose a new therapy target for this disease (Nguyen et al, this issue). ...

journal_title：Science translational medicine

authors： Logothetis C,Aparicio A,Thompson TC

更新日期：2018-05-02 00:00:00

abstract：:Sunitinib malate is a multitargeted receptor tyrosine kinase inhibitor used in the treatment of human malignancies. A substantial number of sunitinib-treated patients develop cardiac dysfunction, but the mechanism of sunitinib-induced cardiotoxicity is poorly understood. We show that mice treated with sunitinib develo...

journal_title：Science translational medicine

authors： Chintalgattu V,Rees ML,Culver JC,Goel A,Jiffar T,Zhang J,Dunner K Jr,Pati S,Bankson JA,Pasqualini R,Arap W,Bryan NS,Taegtmeyer H,Langley RR,Yao H,Kupferman ME,Entman ML,Dickinson ME,Khakoo AY

更新日期：2013-05-29 00:00:00

abstract：:Troublesome involuntary movements in the absence of dopaminergic medication, so-called off-medication dyskinesias, are a serious adverse effect of fetal neural grafts that hinders the development of cell-based therapies for Parkinson's disease. The mechanisms underlying these dyskinesias are not well understood, and i...

journal_title：Science translational medicine

authors： Politis M,Wu K,Loane C,Quinn NP,Brooks DJ,Rehncrona S,Bjorklund A,Lindvall O,Piccini P

更新日期：2010-06-30 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

authors： Bahubeshi A,Tischkowitz M,Foulkes WD

更新日期：2011-11-30 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

authors： Montgomery KL,Iyer SM,Christensen AJ,Deisseroth K,Delp SL

更新日期：2016-05-04 00:00:00

abstract：:Precise form-fitting of prosthetic sockets is important for the comfort and well-being of persons with limb amputations. Capabilities for continuous monitoring of pressure and temperature at the skin-prosthesis interface can be valuable in the fitting process and in monitoring for the development of dangerous regions ...

journal_title：Science translational medicine

authors： Kwak JW,Han M,Xie Z,Chung HU,Lee JY,Avila R,Yohay J,Chen X,Liang C,Patel M,Jung I,Kim J,Namkoong M,Kwon K,Guo X,Ogle C,Grande D,Ryu D,Kim DH,Madhvapathy S,Liu C,Yang DS,Park Y,Caldwell R,Banks A,Xu S,H

更新日期：2020-12-16 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

authors： Roh JH,Huang Y,Bero AW,Kasten T,Stewart FR,Bateman RJ,Holtzman DM

更新日期：2012-09-05 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：: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_title：Science translational medicine

authors： Burkin DJ,Wuebbles RD

更新日期：2012-06-20 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：: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：: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

authors： Mischak H,Allmaier G,Apweiler R,Attwood T,Baumann M,Benigni A,Bennett SE,Bischoff R,Bongcam-Rudloff E,Capasso G,Coon JJ,D'Haese P,Dominiczak AF,Dakna M,Dihazi H,Ehrich JH,Fernandez-Llama P,Fliser D,Frokiaer J,Garin

更新日期：2010-08-25 00:00:00

abstract：:As the prevalence of Alzheimer's disease (AD) grows, so do the costs it imposes on society. Scientific, clinical, and financial interests have focused current drug discovery efforts largely on the single biological pathway that leads to amyloid deposition. This effort has resulted in slow progress and disappointing ou...

journal_title：Science translational medicine

authors： Lo AW,Ho C,Cummings J,Kosik KS

更新日期：2014-06-18 00:00:00

abstract：:Stimulator of interferon genes (STING) is a cytosolic receptor that senses both exogenous and endogenous cytosolic cyclic dinucleotides (CDNs), activating TBK1/IRF3 (interferon regulatory factor 3), NF-κB (nuclear factor κB), and STAT6 (signal transducer and activator of transcription 6) signaling pathways to induce r...

journal_title：Science translational medicine

authors： Fu J,Kanne DB,Leong M,Glickman LH,McWhirter SM,Lemmens E,Mechette K,Leong JJ,Lauer P,Liu W,Sivick KE,Zeng Q,Soares KC,Zheng L,Portnoy DA,Woodward JJ,Pardoll DM,Dubensky TW Jr,Kim Y

更新日期：2015-04-15 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