Emerging viral diseases: confronting threats with new technologies.

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：: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：: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：:Inherited retinal degenerative diseases, a genetically and phenotypically heterogeneous group of disorders, affect the function of photoreceptor cells and are among the leading causes of blindness. Recent advances in molecular genetics and cell biology are elucidating the pathophysiological mechanisms underlying these...

journal_title：Science translational medicine

authors： Scholl HP,Strauss RW,Singh MS,Dalkara D,Roska B,Picaud S,Sahel JA

更新日期：2016-12-07 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：: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：:Glycogen synthase kinase 3 (GSK3), a key regulatory kinase in the wingless-type MMTV integration site family (WNT) pathway, is a therapeutic target of interest in many diseases. Although dual GSK3α/β inhibitors have entered clinical trials, none has successfully translated to clinical application. Mechanism-based toxi...

journal_title：Science translational medicine

authors： Wagner FF,Benajiba L,Campbell AJ,Weïwer M,Sacher JR,Gale JP,Ross L,Puissant A,Alexe G,Conway A,Back M,Pikman Y,Galinsky I,DeAngelo DJ,Stone RM,Kaya T,Shi X,Robers MB,Machleidt T,Wilkinson J,Hermine O,Kung A,St

更新日期：2018-03-07 00:00:00

abstract：:Allergen-specific immunotherapy (AIT) has been used for more than 100 years as a tolerance-inducing therapy for allergic diseases and represents a potentially curative method of treatment. AIT functions through multiple mechanisms, including regulating T and B cell responses, changing antibody isotypes, and decreasing...

journal_title：Science translational medicine

authors： Akdis CA,Akdis M

更新日期：2015-03-25 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：: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：:Measles virus is a highly infectious morbillivirus responsible for major morbidity and mortality in unvaccinated humans. The related, zoonotic canine distemper virus (CDV) induces morbillivirus disease in ferrets with 100% lethality. We report an orally available, shelf-stable pan-morbillivirus inhibitor that targets ...

journal_title：Science translational medicine

authors： Krumm SA,Yan D,Hovingh ES,Evers TJ,Enkirch T,Reddy GP,Sun A,Saindane MT,Arrendale RF,Painter G,Liotta DC,Natchus MG,von Messling V,Plemper RK

更新日期：2014-04-16 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：: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：:The limited predictability of phase II biomarkers for atherosclerosis outcomes in phase III studies stands in contrast to the number and varied types of biomarkers--soluble, imaging, and functional--that have been used in a diverse array of trials. Although collectively abundant, these biomarker data exist in a fragme...

journal_title：Science translational medicine

authors： Fryburg DA,Vassileva MT

更新日期：2011-03-02 00:00:00

abstract：:In recent years, there has been a growing focus on the unreliability of published biomedical and clinical research. To introduce effective new scientific contributors to the culture of health care, we propose a "datathon" or "hackathon" model in which participants with disparate, but potentially synergistic and comple...

journal_title：Science translational medicine

authors： Aboab J,Celi LA,Charlton P,Feng M,Ghassemi M,Marshall DC,Mayaud L,Naumann T,McCague N,Paik KE,Pollard TJ,Resche-Rigon M,Salciccioli JD,Stone DJ

更新日期：2016-04-06 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

authors： Lionel AC,Crosbie J,Barbosa N,Goodale T,Thiruvahindrapuram B,Rickaby J,Gazzellone M,Carson AR,Howe JL,Wang Z,Wei J,Stewart AF,Roberts R,McPherson R,Fiebig A,Franke A,Schreiber S,Zwaigenbaum L,Fernandez BA,Roberts W

更新日期：2011-08-10 00:00:00

abstract：:Advanced ovarian cancer is frequently treated with combination chemotherapy, but high recurrence rates show the need for therapies that can produce durable responses and extend overall survival. Bispecific antibodies that interact with tumor antigens on cancer cells and activating receptors on immune cells offer an in...

journal_title：Science translational medicine

authors： Crawford A,Haber L,Kelly MP,Vazzana K,Canova L,Ram P,Pawashe A,Finney J,Jalal S,Chiu D,Colleton CA,Garnova E,Makonnen S,Hickey C,Krueger P,DelFino F,Potocky T,Kuhnert J,Godin S,Retter MW,Duramad P,MacDonald D,

更新日期：2019-06-19 00:00:00

abstract：:Diabetic patients frequently suffer from continuous pain that is poorly treated by currently available analgesics. We used mouse models of type 1 and type 2 diabetes to investigate a possible role for the hyperpolarization-activated cyclic nucleotide-gated 2 (HCN2) ion channels as drivers of diabetic pain. Blocking or...

journal_title：Science translational medicine

authors： Tsantoulas C,Laínez S,Wong S,Mehta I,Vilar B,McNaughton PA

更新日期：2017-09-27 00:00:00

abstract：:Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) are life-threatening, cutaneous adverse drug reactions that are accompanied by keratinocyte cell death. Dead keratinocytes from SJS/TEN lesions exhibited necrosis, by morphological criteria. Supernatant from peripheral blood mononuclear cells (PBMCs) ...

journal_title：Science translational medicine

authors： Saito N,Qiao H,Yanagi T,Shinkuma S,Nishimura K,Suto A,Fujita Y,Suzuki S,Nomura T,Nakamura H,Nagao K,Obuse C,Shimizu H,Abe R

更新日期：2014-07-16 00:00:00

abstract：:Nipah virus is an emerging pathogen in the Paramyxoviridae family. Upon transmission of Nipah virus from its natural reservoir, Pteropus spp. fruit bats, to humans, it causes respiratory and neurological disease with a case-fatality rate about 70%. Human-to-human transmission has been observed during Nipah virus outbr...

journal_title：Science translational medicine

authors： Lo MK,Feldmann F,Gary JM,Jordan R,Bannister R,Cronin J,Patel NR,Klena JD,Nichol ST,Cihlar T,Zaki SR,Feldmann H,Spiropoulou CF,de Wit E

更新日期：2019-05-29 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：:In humans, exposure to aristolochic acid (AA) is associated with urothelial carcinoma of the upper urinary tract (UTUC). Exome sequencing of UTUCs from 19 individuals with documented exposure to AA revealed a remarkably large number of somatic mutations and an unusual mutational signature attributable to AA. Most of t...

journal_title：Science translational medicine

authors： Hoang ML,Chen CH,Sidorenko VS,He J,Dickman KG,Yun BH,Moriya M,Niknafs N,Douville C,Karchin R,Turesky RJ,Pu YS,Vogelstein B,Papadopoulos N,Grollman AP,Kinzler KW,Rosenquist TA

更新日期：2013-08-07 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：:Regulatory T cells (T(regs)) manipulated ex vivo have potential as cellular therapeutics in autoimmunity and transplantation. Although it is possible to expand naturally occurring T(regs), an attractive alternative possibility, particularly suited to solid organ and bone marrow transplantation, is the stimulation of t...

journal_title：Science translational medicine

authors： Feng G,Nadig SN,Bäckdahl L,Beck S,Francis RS,Schiopu A,Whatcott A,Wood KJ,Bushell A

更新日期：2011-05-18 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

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：: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

authors： Cuende J,Liénart S,Dedobbeleer O,van der Woning B,De Boeck G,Stockis J,Huygens C,Colau D,Somja J,Delvenne P,Hannon M,Baron F,Dumoutier L,Renauld JC,De Haard H,Saunders M,Coulie PG,Lucas S

更新日期：2015-04-22 00:00:00

abstract：:Airway mucus in cystic fibrosis (CF) is highly elastic, but the mechanism behind this pathology is unclear. We hypothesized that the biophysical properties of CF mucus are altered because of neutrophilic oxidative stress. Using confocal imaging, rheology, and biochemical measures of inflammation and oxidation, we foun...

journal_title：Science translational medicine

authors： Yuan S,Hollinger M,Lachowicz-Scroggins ME,Kerr SC,Dunican EM,Daniel BM,Ghosh S,Erzurum SC,Willard B,Hazen SL,Huang X,Carrington SD,Oscarson S,Fahy JV

更新日期：2015-02-25 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：:Venetoclax is a specific B cell lymphoma 2 (BCL-2) inhibitor with promising activity against acute myeloid leukemia (AML), but its clinical efficacy as a single agent or in combination with hypomethylating agents (HMAs), such as azacitidine, is hampered by intrinsic and acquired resistance. Here, we performed a genome...

journal_title：Science translational medicine

authors： Sharon D,Cathelin S,Mirali S,Di Trani JM,Yanofsky DJ,Keon KA,Rubinstein JL,Schimmer AD,Ketela T,Chan SM

更新日期：2019-10-30 00:00:00

abstract：:Variants in the leucine-rich repeat kinase-2 (LRRK2) gene are associated with Parkinson's disease, leprosy, and Crohn's disease, three disorders with inflammation as an important component. Because of its high expression in granulocytes and CD68-positive cells, LRRK2 may have a function in innate immunity. We tested t...

journal_title：Science translational medicine

authors： Shutinoski B,Hakimi M,Harmsen IE,Lunn M,Rocha J,Lengacher N,Zhou YY,Khan J,Nguyen A,Hake-Volling Q,El-Kodsi D,Li J,Alikashani A,Beauchamp C,Majithia J,Coombs K,Shimshek D,Marcogliese PC,Park DS,Rioux JD,Philpott D

更新日期：2019-09-25 00:00:00