Whole-genome sequencing for optimized patient management.

abstract：:Nipah virus (NiV) is an emerging zoonotic paramyxovirus that causes severe and often fatal disease in pigs and humans. There are currently no vaccines or treatments approved for human use. Studies in small-animal models of NiV infection suggest that antibody therapy may be a promising treatment. However, most studies ...

journal_title：Science translational medicine

authors： Geisbert TW,Mire CE,Geisbert JB,Chan YP,Agans KN,Feldmann F,Fenton KA,Zhu Z,Dimitrov DS,Scott DP,Bossart KN,Feldmann H,Broder CC

更新日期：2014-06-25 00:00:00

abstract：:Prolonged life expectancy in the 20th century has been one of humankind's greatest triumphs. However, the substantial increase in the human life span has ushered in a new concern: healthy aging. Because infectious diseases prominently contribute to morbidity in the particularly vulnerable elderly population, strategie...

journal_title：Science translational medicine

authors： Boraschi D,Aguado MT,Dutel C,Goronzy J,Louis J,Grubeck-Loebenstein B,Rappuoli R,Del Giudice G

更新日期：2013-05-15 00:00:00

abstract：:Down syndrome (DS) is among the most frequent genetic causes of intellectual disability, and ameliorating this deficit is a major goal in support of people with trisomy 21. The Ts65Dn mouse recapitulates some major brain structural and behavioral phenotypes of DS, including reduced size and cellularity of the cerebell...

journal_title：Science translational medicine

authors： Das I,Park JM,Shin JH,Jeon SK,Lorenzi H,Linden DJ,Worley PF,Reeves RH

更新日期：2013-09-04 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

authors： Lai D,Chen M,Su J,Liu X,Rothe K,Hu K,Forrest DL,Eaves CJ,Morin GB,Jiang X

更新日期：2018-02-07 00:00:00

abstract：:Axonal dystrophy, indicative of perturbed axonal transport, occurs early during Alzheimer's disease (AD) pathogenesis. Little is known about the mechanisms underlying this initial sign of the pathology. This study proves that Golgi-localized γ-ear-containing ARF binding protein 3 (GGA3) loss of function, due to Gga3 g...

journal_title：Science translational medicine

authors： Lomoio S,Willen R,Kim W,Ho KZ,Robinson EK,Prokopenko D,Kennedy ME,Tanzi RE,Tesco G

更新日期：2020-11-18 00:00:00

abstract：:In populations that have not been selected for family history of disease, it is unclear how commonly pathogenic variants (PVs) in disease-associated genes for rare Mendelian conditions are found and how often they are associated with clinical features of these conditions. We conducted independent, prospective analyses...

journal_title：Science translational medicine

authors： Natarajan P,Gold NB,Bick AG,McLaughlin H,Kraft P,Rehm HL,Peloso GM,Wilson JG,Correa A,Seidman JG,Seidman CE,Kathiresan S,Green RC

更新日期：2016-11-09 00:00:00

abstract：:Despite the high clinical burden, little is known about pathophysiology underlying autism spectrum disorder (ASD). Recent resting-state functional magnetic resonance imaging (rs-fMRI) studies have found atypical synchronization of brain activity in ASD. However, no consensus has been reached on the nature and clinical...

journal_title：Science translational medicine

authors： Holiga Š,Hipp JF,Chatham CH,Garces P,Spooren W,D'Ardhuy XL,Bertolino A,Bouquet C,Buitelaar JK,Bours C,Rausch A,Oldehinkel M,Bouvard M,Amestoy A,Caralp M,Gueguen S,Ly-Le Moal M,Houenou J,Beckmann CF,Loth E,Murphy D

更新日期：2019-02-27 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：:Local delivery of anticancer agents has the potential to maximize treatment efficacy and minimize the acute and long-term systemic toxicities. Here, we used unsupervised systematic evolution of ligands by exponential enrichment to identify four RNA aptamers that specifically recognized mouse and human myeloid cells in...

journal_title：Science translational medicine

authors： De La Fuente A,Zilio S,Caroli J,Van Simaeys D,Mazza EMC,Ince TA,Bronte V,Bicciato S,Weed DT,Serafini P

更新日期：2020-06-17 00:00:00

abstract：:Three-dimensional (3D) titanium-mesh scaffolds offer many advantages over autologous bone grafting for the regeneration of challenging large segmental bone defects. Our study supports the hypothesis that endogenous bone defect regeneration can be promoted by mechanobiologically optimized Ti-mesh scaffolds. Using finit...

journal_title：Science translational medicine

authors： Pobloth AM,Checa S,Razi H,Petersen A,Weaver JC,Schmidt-Bleek K,Windolf M,Tatai AÁ,Roth CP,Schaser KD,Duda GN,Schwabe P

更新日期：2018-01-10 00:00:00

abstract：:Detection of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infections to date has relied heavily on reverse transcription polymerase chain reaction testing. However, limited test availability, high false-negative rates, and the existence of asymptomatic or subclinical infections have resulted in an unde...

journal_title：Science translational medicine

authors： Silverman JD,Hupert N,Washburne AD

更新日期：2020-07-29 00:00:00

abstract：:Limited treatment options exist for patients who suffer from a painful bladder condition known as interstitial cystitis/bladder pain syndrome (IC/BPS). Whether given systemically (orally) or by short-duration (1 to 2 hours) exposure via intravesical instillation, therapeutic agents have exhibited poor efficacy because...

journal_title：Science translational medicine

authors： Nickel JC,Jain P,Shore N,Anderson J,Giesing D,Lee H,Kim G,Daniel K,White S,Larrivee-Elkins C,Lekstrom-Himes J,Cima M

更新日期：2012-07-18 00:00:00

abstract：:MicroRNAs (miRNAs) regulate many aspects of human biology. They target mRNAs for translational repression or degradation through base pairing with 3' untranslated regions, primarily via seed sequences (nucleotides 2 to 8 in the mature miRNA sequence). A number of individual miRNAs and miRNA families share seed sequenc...

journal_title：Science translational medicine

authors： Rottiers V,Obad S,Petri A,McGarrah R,Lindholm MW,Black JC,Sinha S,Goody RJ,Lawrence MS,deLemos AS,Hansen HF,Whittaker S,Henry S,Brookes R,Najafi-Shoushtari SH,Chung RT,Whetstine JR,Gerszten RE,Kauppinen S,Näär AM

更新日期：2013-11-20 00:00:00

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

authors： Gorzelany JA,de Souza MP

更新日期：2013-03-27 00:00:00

abstract：:As observed during the 2013-2016 Ebola virus disease epidemic, containment of filovirus outbreaks is challenging and made more difficult by the lack of approved vaccine or therapeutic options. Marburg and Ravn viruses are highly virulent and cause severe and frequently lethal disease in humans. Monoclonal antibodies (...

journal_title：Science translational medicine

authors： Mire CE,Geisbert JB,Borisevich V,Fenton KA,Agans KN,Flyak AI,Deer DJ,Steinkellner H,Bohorov O,Bohorova N,Goodman C,Hiatt A,Kim DH,Pauly MH,Velasco J,Whaley KJ,Crowe JE Jr,Zeitlin L,Geisbert TW

更新日期：2017-04-05 00:00:00

abstract：:Retinal pigment epithelium (RPE) dysfunction and loss are a hallmark of non-neovascular age-related macular degeneration (NNAMD). Without the RPE, a majority of overlying photoreceptors ultimately degenerate, leading to severe, progressive vision loss. Clinical and histological studies suggest that RPE replacement str...

journal_title：Science translational medicine

authors： Kashani AH,Lebkowski JS,Rahhal FM,Avery RL,Salehi-Had H,Dang W,Lin CM,Mitra D,Zhu D,Thomas BB,Hikita ST,Pennington BO,Johnson LV,Clegg DO,Hinton DR,Humayun MS

更新日期：2018-04-04 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：:Gene therapy is emerging as a therapeutic modality for treating disorders of the retina. Photoreceptor cells are the primary cell type affected in many inherited diseases of retinal degeneration. Successfully treating these diseases with gene therapy requires the identification of efficient and safe targeting vectors ...

journal_title：Science translational medicine

authors： Vandenberghe LH,Bell P,Maguire AM,Cearley CN,Xiao R,Calcedo R,Wang L,Castle MJ,Maguire AC,Grant R,Wolfe JH,Wilson JM,Bennett J

更新日期：2011-06-22 00:00:00

abstract：:Acute myeloid leukemia (AML) is a molecularly and clinically heterogeneous hematological malignancy. Although immunotherapy may be an attractive modality to exploit in patients with AML, the ability to predict the groups of patients and the types of cancer that will respond to immune targeting remains limited. This st...

journal_title：Science translational medicine

authors： Vadakekolathu J,Minden MD,Hood T,Church SE,Reeder S,Altmann H,Sullivan AH,Viboch EJ,Patel T,Ibrahimova N,Warren SE,Arruda A,Liang Y,Smith TH,Foulds GA,Bailey MD,Gowen-MacDonald J,Muth J,Schmitz M,Cesano A,Pockley

更新日期：2020-06-03 00:00:00

abstract：:Osteoclasts actively remodel both the mineral and proteinaceous components of bone during normal growth and development as well as pathologic states ranging from osteoporosis to bone metastasis. The cysteine proteinase cathepsin K confers osteoclasts with potent type I collagenolytic activity; however, cathepsin K-nul...

journal_title：Science translational medicine

authors： Zhu L,Tang Y,Li XY,Keller ET,Yang J,Cho JS,Feinberg TY,Weiss SJ

更新日期：2020-02-05 00:00:00

abstract：:Interpandemic or seasonal influenza A, currently subtypes H3N2 and H1N1, exacts an enormous annual burden both in terms of human health and economic impact. Incidence prediction ahead of season remains a challenge largely because of the virus' antigenic evolution. We propose a forecasting approach that incorporates ev...

journal_title：Science translational medicine

authors： Du X,King AA,Woods RJ,Pascual M

更新日期：2017-10-25 00:00:00

abstract：:An innovative strategy for delivering drugs to chondrocytes in situ offers new avenues for treating osteoarthritis (Geiger et al, this issue). ...

journal_title：Science translational medicine

authors： Evans CH

更新日期：2018-11-28 00:00:00

abstract：:Clinical diagnostic tools requiring direct sample testing cannot be applied to infections deep within the body, and clinically available imaging tools lack specificity. New approaches are needed for early diagnosis and monitoring of bacterial infections and rapid detection of drug-resistant organisms. Molecular imagin...

journal_title：Science translational medicine

authors： Ordonez AA,Sellmyer MA,Gowrishankar G,Ruiz-Bedoya CA,Tucker EW,Palestro CJ,Hammoud DA,Jain SK

更新日期：2019-09-04 00:00:00

abstract：:The glucagon-like peptide-1 receptor (GLP-1R) agonist liraglutide is approved for the treatment of obesity; however, there is still much to be learned regarding the neuronal sites of action that underlie its suppressive effects on food intake and body weight. Peripherally administered liraglutide in rats acts in part ...

journal_title：Science translational medicine

authors： Fortin SM,Lipsky RK,Lhamo R,Chen J,Kim E,Borner T,Schmidt HD,Hayes MR

更新日期：2020-03-04 00:00:00

abstract：:Amine-containing polymers immobilized on mesh and placed at trauma sites scavenge biomolecules that initiate a damaging immune response. ...

journal_title：Science translational medicine

authors： Ulery BD

更新日期：2017-02-22 00:00:00

abstract：:Cancer immunotherapy using antibodies that target immune checkpoints has delivered outstanding results. However, responses only occur in a subset of patients, and it is not fully understood what biological processes determine an effective outcome. This lack of understanding hinders the development of rational combinat...

journal_title：Science translational medicine

authors： Zemek RM,De Jong E,Chin WL,Schuster IS,Fear VS,Casey TH,Forbes C,Dart SJ,Leslie C,Zaitouny A,Small M,Boon L,Forrest ARR,Muiri DO,Degli-Esposti MA,Millward MJ,Nowak AK,Lassmann T,Bosco A,Lake RA,Lesterhuis WJ

更新日期：2019-07-17 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

authors： Tang AT,Sullivan KR,Hong CC,Goddard LM,Mahadevan A,Ren A,Pardo H,Peiper A,Griffin E,Tanes C,Mattei LM,Yang J,Li L,Mericko-Ishizuka P,Shen L,Hobson N,Girard R,Lightle R,Moore T,Shenkar R,Polster SP,Roedel CJ,Li

更新日期：2019-11-27 00:00:00

abstract：:Parenteral and oral routes have been the traditional methods of administering cytotoxic agents to cancer patients. Unfortunately, the maximum potential effect of these cytotoxic agents has been limited because of systemic toxicity and poor tumor perfusion. In an attempt to improve the efficacy of cytotoxic agents whil...

journal_title：Science translational medicine

authors： Byrne JD,Jajja MR,O'Neill AT,Bickford LR,Keeler AW,Hyder N,Wagner K,Deal A,Little RE,Moffitt RA,Stack C,Nelson M,Brooks CR,Lee W,Luft JC,Napier ME,Darr D,Anders CK,Stack R,Tepper JE,Wang AZ,Zamboni WC,Yeh JJ,

更新日期：2015-02-04 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：: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

authors： Ter Horst KW,Lammers NM,Trinko R,Opland DM,Figee M,Ackermans MT,Booij J,van den Munckhof P,Schuurman PR,Fliers E,Denys D,DiLeone RJ,la Fleur SE,Serlie MJ

更新日期：2018-05-23 00:00:00