Sponsors meet scientists to speed pediatric medicines development.

abstract：:In multiple sclerosis (MS), lymphocyte--in particular B cell--transit between the central nervous system (CNS) and periphery may contribute to the maintenance of active disease. Clonally related B cells exist in the cerebrospinal fluid (CSF) and peripheral blood (PB) of MS patients; however, it remains unclear which s...

journal_title：Science translational medicine

authors： Palanichamy A,Apeltsin L,Kuo TC,Sirota M,Wang S,Pitts SJ,Sundar PD,Telman D,Zhao LZ,Derstine M,Abounasr A,Hauser SL,von Büdingen HC

更新日期：2014-08-06 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

authors： Dietz HC

更新日期：2015-05-20 00:00:00

abstract：:Central tolerance plays a key role in modulating immune responses to self and exogenous antigens. The absence of self-antigen expression, as in patients with genetic deficiencies, prevents the development of antigen-specific immune tolerance. Hence, a substantial number of patients develop neutralizing antibodies to t...

journal_title：Science translational medicine

authors： Gupta N,Culina S,Meslier Y,Dimitrov J,Arnoult C,Delignat S,Gangadharan B,Lecerf M,Justesen S,Gouilleux-Gruart V,Salomon BL,Scott DW,Kaveri SV,Mallone R,Lacroix-Desmazes S

更新日期：2015-02-18 00:00:00

abstract：:Amyotrophic lateral sclerosis (ALS), a fatal disease causing progressive loss of motor neurons, still has no effective treatment. We developed a phenotypic screen to repurpose existing drugs using ALS motor neuron survival as readout. Motor neurons were generated from induced pluripotent stem cells (iPSCs) derived fro...

journal_title：Science translational medicine

authors： Imamura K,Izumi Y,Watanabe A,Tsukita K,Woltjen K,Yamamoto T,Hotta A,Kondo T,Kitaoka S,Ohta A,Tanaka A,Watanabe D,Morita M,Takuma H,Tamaoka A,Kunath T,Wray S,Furuya H,Era T,Makioka K,Okamoto K,Fujisawa T,Nishit

更新日期：2017-05-24 00:00:00

abstract：:Lung cancer is more deadly than colon, breast, and prostate cancers combined, and treatment improvements have failed to improve prognosis significantly. Here, we identify a critical mediator of lung cancer progression, Rac1b, a tumor-associated protein with cell-transforming properties that are linked to the matrix me...

journal_title：Science translational medicine

authors： Stallings-Mann ML,Waldmann J,Zhang Y,Miller E,Gauthier ML,Visscher DW,Downey GP,Radisky ES,Fields AP,Radisky DC

更新日期：2012-07-11 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

authors： Friedman CP,Wong AK,Blumenthal D

更新日期：2010-11-10 00:00:00

abstract：:Tuberculous meningitis (TBM) is a devastating form of tuberculosis (TB), and key TB antimicrobials, including rifampin, have restricted brain penetration. A lack of reliable data on intralesional drug biodistribution in infected tissues has limited pharmacokinetic (PK) modeling efforts to optimize TBM treatments. Curr...

journal_title：Science translational medicine

authors： Tucker EW,Guglieri-Lopez B,Ordonez AA,Ritchie B,Klunk MH,Sharma R,Chang YS,Sanchez-Bautista J,Frey S,Lodge MA,Rowe SP,Holt DP,Gobburu JVS,Peloquin CA,Mathews WB,Dannals RF,Pardo CA,Kannan S,Ivaturi VD,Jain SK

更新日期：2018-12-05 00:00:00

abstract：:Long synthetic peptides (LSPs) have a variety of important clinical uses as synthetic vaccines and drugs. Techniques for peptide synthesis were revolutionized in the 1960s and 1980s, after which efficient techniques for purification and characterization of the product were developed. These improved techniques allowed ...

journal_title：Science translational medicine

authors： Corradin G,Kajava AV,Verdini A

更新日期：2010-09-22 00:00:00

abstract：:The neurotransmitter glutamate is the primary excitatory neurotransmitter in mammalian brain and is responsible for most corticocortical and corticofugal neurotransmission. Disturbances in glutamatergic function have been implicated in the pathophysiology of several neuropsychiatric disorders-including schizophrenia, ...

journal_title：Science translational medicine

authors： Javitt DC,Schoepp D,Kalivas PW,Volkow ND,Zarate C,Merchant K,Bear MF,Umbricht D,Hajos M,Potter WZ,Lee CM

更新日期：2011-09-28 00:00:00

abstract：:Retinal prosthetic implants are the only approved treatment for retinitis pigmentosa, a disease of the eye that causes blindness through gradual degeneration of photoreceptors. An array of microelectrodes triggered by input from a camera stimulates surviving retinal neurons, with each electrode acting as a pixel. Unin...

journal_title：Science translational medicine

authors： Weitz AC,Nanduri D,Behrend MR,Gonzalez-Calle A,Greenberg RJ,Humayun MS,Chow RH,Weiland JD

更新日期：2015-12-16 00:00:00

abstract：:Translation in an academic environment requires a support system--people, goals, models, partnerships, and infrastructures--that will push promising basic science and technology projects forward into the clinic. ...

journal_title：Science translational medicine

authors： Volk HD,Stevens MM,Mooney DJ,Grainger DW,Duda GN

更新日期：2015-04-08 00:00:00

abstract：:Platelets are crucial for normal hemostasis; however, their hyperactivation also contributes to many potentially lethal pathologies including myocardial infarction, stroke, and cancer. We hypothesized that modified platelets lacking their aggregation and activation capacity could act as reversible inhibitors of platel...

journal_title：Science translational medicine

authors： Papa AL,Jiang A,Korin N,Chen MB,Langan ET,Waterhouse A,Nash E,Caroff J,Graveline A,Vernet A,Mammoto A,Mammoto T,Jain A,Kamm RD,Gounis MJ,Ingber DE

更新日期：2019-02-13 00:00:00

abstract：:Mycosis fungoides (MF), the most common cutaneous T cell lymphoma (CTCL) is a malignancy of skin-tropic memory T cells. Most MF cases present as early stage (stage I A/B, limited to the skin), and these patients typically have a chronic, indolent clinical course. However, a small subset of early-stage cases develop pr...

journal_title：Science translational medicine

authors： de Masson A,O'Malley JT,Elco CP,Garcia SS,Divito SJ,Lowry EL,Tawa M,Fisher DC,Devlin PM,Teague JE,Leboeuf NR,Kirsch IR,Robins H,Clark RA,Kupper TS

更新日期：2018-05-09 00:00:00

abstract：:A clinical trial evaluating HIV vaccine therapy teaches us much about optimal design (Sneller et al, this issue). ...

journal_title：Science translational medicine

authors： Michael NL

更新日期：2017-12-06 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：:Currently, no vaccine exists for hepatitis C virus (HCV), a major pathogen thought to infect 170 million people globally. Many studies suggest that host T cell responses are critical for spontaneous resolution of disease, and preclinical studies have indicated a requirement for T cells in protection against challenge....

journal_title：Science translational medicine

authors： Barnes E,Folgori A,Capone S,Swadling L,Aston S,Kurioka A,Meyer J,Huddart R,Smith K,Townsend R,Brown A,Antrobus R,Ammendola V,Naddeo M,O'Hara G,Willberg C,Harrison A,Grazioli F,Esposito ML,Siani L,Traboni C,Oo Y

更新日期：2012-01-04 00:00:00

abstract：:Patients with rheumatoid arthritis (RA) may display atypical CD21-/lo B cells in their blood, but the implication of this observation remains unclear. We report here that the group of patients with RA and elevated frequencies of CD21-/lo B cells shows decreased ataxia telangiectasia-mutated (ATM) expression and activa...

journal_title：Science translational medicine

authors： Mensah KA,Chen JW,Schickel JN,Isnardi I,Yamakawa N,Vega-Loza A,Anolik JH,Gatti RA,Gelfand EW,Montgomery RR,Horowitz MC,Craft JE,Meffre E

更新日期：2019-11-20 00:00:00

abstract：:Promising therapies for cartilage repair are translated through large animal models toward human application. To guide this work, regulatory agencies publish recommendations ("guidance documents") to direct pivotal large animal studies. These are meant to aid in study design, outline metrics for judging efficacy, and ...

journal_title：Science translational medicine

authors： Pfeifer CG,Fisher MB,Carey JL,Mauck RL

更新日期：2015-10-21 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

authors： Potter R,Patterson BW,Elbert DL,Ovod V,Kasten T,Sigurdson W,Mawuenyega K,Blazey T,Goate A,Chott R,Yarasheski KE,Holtzman DM,Morris JC,Benzinger TL,Bateman RJ

更新日期：2013-06-12 00:00:00

abstract：:Adding an electrical pulse to a microfluidic device that squeezes cells through narrow channels efficiently delivers DNA to the nucleus. ...

journal_title：Science translational medicine

authors： King KR

更新日期：2017-03-22 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：:Therapeutic intravesical instillation of bacillus Calmette-Guérin (BCG) is effective at triggering inflammation and eliciting successful tumor immunity in patients with non-muscle invasive bladder cancer, with 50 to 70% clinical response. Therapeutic success relies on repeated instillations of live BCG administered as...

journal_title：Science translational medicine

authors： Biot C,Rentsch CA,Gsponer JR,Birkhäuser FD,Jusforgues-Saklani H,Lemaître F,Auriau C,Bachmann A,Bousso P,Demangel C,Peduto L,Thalmann GN,Albert ML

更新日期：2012-06-06 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

authors： Wu MH,Ma WL,Hsu CL,Chen YL,Ou JH,Ryan CK,Hung YC,Yeh S,Chang C

更新日期：2010-05-19 00:00:00

abstract：:Conventional radiation therapy for cancer usually consists of multiple treatments (called fractions) with low doses of radiation. These dose schemes are planned with the guidance of the linear-quadratic (LQ) model, which has been the most prevalent model for designing dose schemes in radiation therapy. The high-dose f...

journal_title：Science translational medicine

authors： Wang JZ,Huang Z,Lo SS,Yuh WT,Mayr NA

更新日期：2010-07-07 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

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：: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：: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

authors： Sondhi D,Kaminsky SM,Hackett NR,Pagovich OE,Rosenberg JB,De BP,Chen A,Van de Graaf B,Mezey JG,Mammen GW,Mancenido D,Xu F,Kosofsky B,Yohay K,Worgall S,Kaner RJ,Souwedaine M,Greenwald BM,Kaplitt M,Dyke JP,Ballon DJ

更新日期：2020-12-02 00:00:00

abstract：:Cancer-associated thrombocytosis and high concentrations of circulating transforming growth factor-β1 (TGF-β1) are frequently observed in patients with progressive cancers. Using genetic and pharmacological approaches, we show a direct link between thrombin catalytic activity and release of mature TGF-β1 from platelet...

journal_title：Science translational medicine

authors： Metelli A,Wu BX,Riesenberg B,Guglietta S,Huck JD,Mills C,Li A,Rachidi S,Krieg C,Rubinstein MP,Gewirth DT,Sun S,Lilly MB,Wahlquist AH,Carbone DP,Yang Y,Liu B,Li Z

更新日期：2020-01-08 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

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