The study is open: Participants are now recruiting investigators.

abstract：:Common variable immune deficiency (CVID) is the most common symptomatic primary immune deficiency, affecting ~1 in 25,000 persons. These patients suffer from impaired antibody responses, autoimmunity, and susceptibility to lymphoid cancers. To explore the cellular basis for these clinical phenotypes, we conducted high...

journal_title：Science translational medicine

authors： Roskin KM,Simchoni N,Liu Y,Lee JY,Seo K,Hoh RA,Pham T,Park JH,Furman D,Dekker CL,Davis MM,James JA,Nadeau KC,Cunningham-Rundles C,Boyd SD

更新日期：2015-08-26 00:00:00

abstract：:Vaccines directed against the blood stages of Plasmodium falciparum malaria are intended to prevent the parasite from invading and replicating within host cells. No blood-stage malaria vaccine has shown clinical efficacy in humans. Most malaria vaccine antigens are parasite surface proteins that have evolved extensive...

journal_title：Science translational medicine

authors： Takala SL,Coulibaly D,Thera MA,Batchelor AH,Cummings MP,Escalante AA,Ouattara A,Traoré K,Niangaly A,Djimdé AA,Doumbo OK,Plowe CV

更新日期：2009-10-14 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：: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：: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：: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：: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：: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：:Sustained viremia after acute HIV infection is associated with profound CD4+ T cell loss and exhaustion of HIV-specific CD8+ T cell responses. To determine the impact of combination antiretroviral therapy (cART) on these processes, we examined the evolution of immune responses in acutely infected individuals initiatin...

journal_title：Science translational medicine

authors： Ndhlovu ZM,Kazer SW,Nkosi T,Ogunshola F,Muema DM,Anmole G,Swann SA,Moodley A,Dong K,Reddy T,Brockman MA,Shalek AK,Ndung'u T,Walker BD

更新日期：2019-05-22 00:00:00

abstract：:A skin-penetrant small molecule activates melanin production to protect cells from cancer-inducing ultraviolet damage. ...

journal_title：Science translational medicine

authors： Venere M

更新日期：2017-07-05 00:00:00

abstract：:Much of our knowledge of the mechanisms underlying plasticity in the visual cortex in response to visual impairment, vision restoration, and environmental interactions comes from animal studies. We evaluated human brain plasticity in a group of patients with Leber's congenital amaurosis (LCA), who regained vision thro...

journal_title：Science translational medicine

authors： Ashtari M,Zhang H,Cook PA,Cyckowski LL,Shindler KS,Marshall KA,Aravand P,Vossough A,Gee JC,Maguire AM,Baker CI,Bennett J

更新日期：2015-07-15 00:00:00

abstract：:Targeting androgen receptor (AR) axis signaling by disrupting androgen-AR interactions remains the primary treatment for metastatic prostate cancer. Unfortunately, all men develop resistance to primary castrating therapy and secondary androgen deprivation therapies (ADTs). Resistance develops in part because castratio...

journal_title：Science translational medicine

authors： Schweizer MT,Antonarakis ES,Wang H,Ajiboye AS,Spitz A,Cao H,Luo J,Haffner MC,Yegnasubramanian S,Carducci MA,Eisenberger MA,Isaacs JT,Denmeade SR

更新日期：2015-01-07 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

authors： Investigators of the US–Latin America Cancer Research Network.

更新日期：2015-12-23 00:00:00

abstract：:Some funding agencies now require consideration of sex and gender in preclinical research, a policy that heralds opportunities and challenges for researchers. ...

journal_title：Science translational medicine

authors： Danska JS

更新日期：2014-10-15 00:00:00

abstract：:Preclinical data suggest the possibility of repurposing a drug for early intervention in envenoming by snake venom rich in metalloproteinases (Albulescu et al., this issue). ...

journal_title：Science translational medicine

authors： Koh CY,Bendre R,Kini RM

更新日期：2020-05-06 00:00:00

abstract：:Tumor escape from immune-mediated destruction has been associated with immunosuppressive mechanisms that inhibit T cell activation. Although evidence for an active immune response, including infiltration with CD8(+) T cells, can be found in a subset of patients, those tumors are nonetheless not immunologically rejecte...

journal_title：Science translational medicine

authors： Spranger S,Spaapen RM,Zha Y,Williams J,Meng Y,Ha TT,Gajewski TF

更新日期：2013-08-28 00:00:00

abstract：:Glucagon-like peptide-1 receptor (GLP1R) agonists and dipeptidyl peptidase 4 inhibitors are widely prescribed diabetes drugs due to their ability to stimulate insulin secretion from remaining β cells and to reduce caloric intake. Unfortunately, they fail to increase human β cell proliferation. Small-molecule inhibitor...

journal_title：Science translational medicine

authors： Ackeifi C,Wang P,Karakose E,Manning Fox JE,González BJ,Liu H,Wilson J,Swartz E,Berrouet C,Li Y,Kumar K,MacDonald PE,Sanchez R,Thorens B,DeVita R,Homann D,Egli D,Scott DK,Garcia-Ocaña A,Stewart AF

更新日期：2020-02-12 00:00:00

abstract：:The Commentary of Mills and Sykes in Science Translational Medicine presented their thesis on the advent of high-throughput technologies and the dangers they may represent for the future of biomedical research. In response, we argue that true progress on the diagnosis and treatment of common human diseases will requir...

journal_title：Science translational medicine

authors： Lamb JR,Gibson NW

更新日期：2010-03-31 00:00:00

abstract：:Nonalcoholic fatty liver disease (NAFLD) is estimated to affect up to one-third of the general population, and new therapies are urgently required. Our laboratory previously developed a controlled-release mitochondrial protonophore (CRMP) that is functionally liver-targeted and promotes oxidation of hepatic triglyceri...

journal_title：Science translational medicine

authors： Goedeke L,Peng L,Montalvo-Romeral V,Butrico GM,Dufour S,Zhang XM,Perry RJ,Cline GW,Kievit P,Chng K,Petersen KF,Shulman GI

更新日期：2019-10-02 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：:Case fatality rates for severe malaria remain high even in the best clinical settings because antimalarial drugs act against the parasite without alleviating life-threatening inflammation. We assessed the potential for host-directed therapy of severe malaria of a new class of anti-inflammatory drugs, the innate defens...

journal_title：Science translational medicine

authors： Achtman AH,Pilat S,Law CW,Lynn DJ,Janot L,Mayer ML,Ma S,Kindrachuk J,Finlay BB,Brinkman FS,Smyth GK,Hancock RE,Schofield L

更新日期：2012-05-23 00:00:00

abstract：:Merkel cell polyomavirus (MCV) causes ~80% of primary and metastatic Merkel cell carcinomas (MCCs). By comparing digital transcriptome subtraction deep-sequencing profiles, we found that transcripts of the cellular survivin oncoprotein [BIRC5a (baculoviral inhibitor of apoptosis repeat-containing 5)] were up-regulated...

journal_title：Science translational medicine

authors： Arora R,Shuda M,Guastafierro A,Feng H,Toptan T,Tolstov Y,Normolle D,Vollmer LL,Vogt A,Dömling A,Brodsky JL,Chang Y,Moore PS

更新日期：2012-05-09 00:00:00

abstract：:Preexisting lymphocytic infiltration of tumors is associated with superior prognostic outcomes in a variety of cancers. Recent studies also suggest that lymphocytic responses may identify patients more likely to benefit from therapies targeting immune checkpoints, suggesting that therapeutic efficacy of immune checkpo...

journal_title：Science translational medicine

authors： Zamarin D,Holmgaard RB,Subudhi SK,Park JS,Mansour M,Palese P,Merghoub T,Wolchok JD,Allison JP

更新日期：2014-03-05 00:00:00

abstract：:We tested the hypothesis that ex vivo hepatocyte gene therapy can correct the metabolic disorder in fumarylacetoacetate hydrolase-deficient (Fah(-/-)) pigs, a large animal model of hereditary tyrosinemia type 1 (HT1). Recipient Fah(-/-) pigs underwent partial liver resection and hepatocyte isolation by collagenase dig...

journal_title：Science translational medicine

authors： Hickey RD,Mao SA,Glorioso J,Elgilani F,Amiot B,Chen H,Rinaldo P,Marler R,Jiang H,DeGrado TR,Suksanpaisan L,O'Connor MK,Freeman BL,Ibrahim SH,Peng KW,Harding CO,Ho CS,Grompe M,Ikeda Y,Lillegard JB,Russell SJ,Nybe

更新日期：2016-07-27 00:00:00

abstract：:Sepsis remains a major public health problem with no major therapeutic advances over the last several decades. The clinical and biological heterogeneity of sepsis have limited success of potential new therapies. Accordingly, there is considerable interest in developing a precision medicine approach to inform more rati...

journal_title：Science translational medicine

authors： Wong HR,Caldwell JT,Cvijanovich NZ,Weiss SL,Fitzgerald JC,Bigham MT,Jain PN,Schwarz A,Lutfi R,Nowak J,Allen GL,Thomas NJ,Grunwell JR,Baines T,Quasney M,Haileselassie B,Lindsell CJ

更新日期：2019-11-13 00:00:00

abstract：:Advances in health information technology and electronic medical records have the tremendous potential to accelerate translational and clinical research. However, privacy concerns threaten to be a rate-limiting factor. By recognizing and responding to patient privacy concerns, policy-makers, researchers, and informati...

journal_title：Science translational medicine

authors： Shelton RH

更新日期：2011-02-09 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

authors： Dakin SG,Martinez FO,Yapp C,Wells G,Oppermann U,Dean BJ,Smith RD,Wheway K,Watkins B,Roche L,Carr AJ

更新日期：2015-10-28 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：:Patients undergoing surgical resection of primary breast tumors confront a risk for metastatic recurrence that peaks sharply 12 to 18 months after surgery. The cause of early metastatic relapse in breast cancer has long been debated, with many ascribing these relapses to the natural progression of the disease. Others ...

journal_title：Science translational medicine

authors： Krall JA,Reinhardt F,Mercury OA,Pattabiraman DR,Brooks MW,Dougan M,Lambert AW,Bierie B,Ploegh HL,Dougan SK,Weinberg RA

更新日期：2018-04-11 00:00:00

abstract：:Physiological data are routinely recorded in intensive care, but their use for rapid assessment of illness severity or long-term morbidity prediction has been limited. We developed a physiological assessment score for preterm newborns, akin to an electronic Apgar score, based on standard signals recorded noninvasively...

journal_title：Science translational medicine

authors： Saria S,Rajani AK,Gould J,Koller D,Penn AA

更新日期：2010-09-08 00:00:00