Clinical trial design: The nobility of randomization.

Abstract:

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

journal_name

Sci Transl Med

authors

Michael NL

doi

10.1126/scitranslmed.aaq0810

subject

Has Abstract

pub_date

2017-12-06 00:00:00

issue

419

eissn

1946-6234

issn

1946-6242

pii

9/419/eaaq0810

journal_volume

9

pub_type

杂志文章,评审
  • A light-reflecting balloon catheter for atraumatic tissue defect repair.

    abstract::A congenital or iatrogenic tissue defect often requires closure by open surgery or metallic components that can erode tissue. Biodegradable, hydrophobic light-activated adhesives represent an attractive alternative to sutures, but lack a specifically designed minimally invasive delivery tool, which limits their clinic...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaa2406

    authors: Roche ET,Fabozzo A,Lee Y,Polygerinos P,Friehs I,Schuster L,Whyte W,Casar Berazaluce AM,Bueno A,Lang N,Pereira MJ,Feins E,Wasserman S,O'Cearbhaill ED,Vasilyev NV,Mooney DJ,Karp JM,Del Nido PJ,Walsh CJ

    更新日期:2015-09-23 00:00:00

  • Wireless sensors for continuous, multimodal measurements at the skin interface with lower limb prostheses.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.abc4327

    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

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

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaf3838

    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

  • PP2A inhibition sensitizes cancer stem cells to ABL tyrosine kinase inhibitors in BCR-ABL+ human leukemia.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aan8735

    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

  • Noninvasive high-resolution electromyometrial imaging of uterine contractions in a translational sheep model.

    abstract::In current clinical practice, uterine contractions are monitored via a tocodynamometer or an intrauterine pressure catheter, both of which provide crude information about contractions. Although electrohysterography/electromyography can measure uterine electrical activity, this method lacks spatial specificity and thus...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aau1428

    authors: Wu W,Wang H,Zhao P,Talcott M,Lai S,McKinstry RC,Woodard PK,Macones GA,Schwartz AL,Cahill AG,Cuculich PS,Wang Y

    更新日期:2019-03-13 00:00:00

  • Acute kidney injury promotes development of papillary renal cell adenoma and carcinoma from renal progenitor cells.

    abstract::Acute tissue injury causes DNA damage and repair processes involving increased cell mitosis and polyploidization, leading to cell function alterations that may potentially drive cancer development. Here, we show that acute kidney injury (AKI) increased the risk for papillary renal cell carcinoma (pRCC) development and...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaw6003

    authors: Peired AJ,Antonelli G,Angelotti ML,Allinovi M,Guzzi F,Sisti A,Semeraro R,Conte C,Mazzinghi B,Nardi S,Melica ME,De Chiara L,Lazzeri E,Lasagni L,Lottini T,Landini S,Giglio S,Mari A,Di Maida F,Antonelli A,Porpiglia F

    更新日期:2020-03-25 00:00:00

  • Regulation of immune responses to protein therapeutics by transplacental induction of T cell tolerance.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaa1957

    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

  • Transcriptome analysis of GVHD reveals aurora kinase A as a targetable pathway for disease prevention.

    abstract::Graft-versus-host disease (GVHD) is the most common complication of hematopoietic stem cell transplant (HCT). However, our understanding of the molecular pathways that cause this disease remains incomplete, leading to inadequate treatment strategies. To address this, we measured the gene expression profile of nonhuman...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aad3231

    authors: Furlan SN,Watkins B,Tkachev V,Flynn R,Cooley S,Ramakrishnan S,Singh K,Giver C,Hamby K,Stempora L,Garrett A,Chen J,Betz KM,Ziegler CG,Tharp GK,Bosinger SE,Promislow DE,Miller JS,Waller EK,Blazar BR,Kean LS

    更新日期:2015-11-25 00:00:00

  • Parallel discovery of Alzheimer's therapeutics.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3008228

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

    更新日期:2014-06-18 00:00:00

  • Grease fires turn up the heat in autoimmune disease.

    abstract::Restoring reverse cholesterol transport ameliorates the B cell hyperexpansion and autoimmune disease found to be associated with accumulation of cholesterol in CD11c+ antigen-presenting cells. ...

    journal_title:Science translational medicine

    pub_type: 评论,杂志文章

    doi:10.1126/scitranslmed.aal4997

    authors: Bhakta NR

    更新日期:2017-01-25 00:00:00

  • Structural, biochemical, and clinical characterization of epidermal growth factor receptor (EGFR) exon 20 insertion mutations in lung cancer.

    abstract::Epidermal growth factor receptor (EGFR) gene mutations (G719X, exon 19 deletions/insertions, L858R, and L861Q) predict favorable responses to EGFR tyrosine kinase inhibitors (TKIs) in advanced non-small cell lung cancer (NSCLC). However, EGFR exon 20 insertion mutations (~10% of all EGFR mutations) are generally assoc...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3007205

    authors: Yasuda H,Park E,Yun CH,Sng NJ,Lucena-Araujo AR,Yeo WL,Huberman MS,Cohen DW,Nakayama S,Ishioka K,Yamaguchi N,Hanna M,Oxnard GR,Lathan CS,Moran T,Sequist LV,Chaft JE,Riely GJ,Arcila ME,Soo RA,Meyerson M,Eck MJ,K

    更新日期:2013-12-18 00:00:00

  • Leukotriene B4 antagonism ameliorates experimental lymphedema.

    abstract::Acquired lymphedema is a cancer sequela and a global health problem currently lacking pharmacologic therapy. We have previously demonstrated that ketoprofen, an anti-inflammatory agent with dual 5-lipoxygenase and cyclooxygenase inhibitory properties, effectively reverses histopathology in experimental lymphedema. We ...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aal3920

    authors: Tian W,Rockson SG,Jiang X,Kim J,Begaye A,Shuffle EM,Tu AB,Cribb M,Nepiyushchikh Z,Feroze AH,Zamanian RT,Dhillon GS,Voelkel NF,Peters-Golden M,Kitajewski J,Dixon JB,Nicolls MR

    更新日期:2017-05-10 00:00:00

  • Immune-mediated pathology in Duchenne muscular dystrophy.

    abstract::Immunological and inflammatory processes downstream of dystrophin deficiency as well as metabolic abnormalities, defective autophagy, and loss of regenerative capacity all contribute to muscle pathology in Duchenne muscular dystrophy (DMD). These downstream cascades offer potential avenues for pharmacological interven...

    journal_title:Science translational medicine

    pub_type: 杂志文章,评审

    doi:10.1126/scitranslmed.aaa7322

    authors: Rosenberg AS,Puig M,Nagaraju K,Hoffman EP,Villalta SA,Rao VA,Wakefield LM,Woodcock J

    更新日期:2015-08-05 00:00:00

  • Tmc gene therapy restores auditory function in deaf mice.

    abstract::Genetic hearing loss accounts for up to 50% of prelingual deafness worldwide, yet there are no biologic treatments currently available. To investigate gene therapy as a potential biologic strategy for restoration of auditory function in patients with genetic hearing loss, we tested a gene augmentation approach in mous...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aab1996

    authors: Askew C,Rochat C,Pan B,Asai Y,Ahmed H,Child E,Schneider BL,Aebischer P,Holt JR

    更新日期:2015-07-08 00:00:00

  • Advances in allergen immunotherapy: aiming for complete tolerance to allergens.

    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

    pub_type: 杂志文章,评审

    doi:10.1126/scitranslmed.aaa7390

    authors: Akdis CA,Akdis M

    更新日期:2015-03-25 00:00:00

  • Stopping sepsis by targeting sphingosine kinase 1.

    abstract::Each year, more than a half million people in the United States alone die from sepsis, a dire multisystem disease with highly inadequate treatment options. In a recent issue of Science, Puneet and colleagues provide compelling evidence that inhibiting sphingosine kinase 1--an enzyme that resides in immune cells and is...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3001299

    authors: O'Neill LA

    更新日期:2010-06-16 00:00:00

  • Lipotoxicity causes multisystem organ failure and exacerbates acute pancreatitis in obesity.

    abstract::Obesity increases the risk of adverse outcomes during acute critical illnesses such as burns, severe trauma, and acute pancreatitis. Although individuals with more body fat and higher serum cytokines and lipase are more likely to experience problems, the roles that these characteristics play are not clear. We used sev...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3002573

    authors: Navina S,Acharya C,DeLany JP,Orlichenko LS,Baty CJ,Shiva SS,Durgampudi C,Karlsson JM,Lee K,Bae KT,Furlan A,Behari J,Liu S,McHale T,Nichols L,Papachristou GI,Yadav D,Singh VP

    更新日期:2011-11-02 00:00:00

  • High-throughput sequencing of the T cell receptor β gene identifies aggressive early-stage mycosis fungoides.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aar5894

    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

  • Combination of an oncolytic virus with PD-L1 blockade keeps cancer in check.

    abstract::An oncolytic vaccinia virus expressing CXCL11 combined with PD-L1 blockade significantly reduces tumor burden and improves survival in murine cancer models. ...

    journal_title:Science translational medicine

    pub_type: 评论,杂志文章

    doi:10.1126/scitranslmed.aan2781

    authors: Jonas BA

    更新日期:2017-04-19 00:00:00

  • Therapy with CTLA4-Ig and an antiviral monoclonal antibody controls chikungunya virus arthritis.

    abstract::In 2013, chikungunya virus (CHIKV) transmission was documented in the Western Hemisphere, and the virus has since spread throughout the Americas with more than 1.8 million people infected in more than 40 countries. CHIKV targets the joints, resulting in symmetric polyarthritis that clinically mimics rheumatoid arthrit...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aah3438

    authors: Miner JJ,Cook LE,Hong JP,Smith AM,Richner JM,Shimak RM,Young AR,Monte K,Poddar S,Crowe JE Jr,Lenschow DJ,Diamond MS

    更新日期:2017-02-01 00:00:00

  • Plasmacytoid dendritic cells, interferon signaling, and FcγR contribute to pathogenesis and therapeutic response in childhood immune thrombocytopenia.

    abstract::Immune thrombocytopenia (ITP) is an autoimmune disorder of childhood characterized by immune-mediated destruction of platelets. The mechanisms underlying the pathogenesis of ITP and the therapeutic efficacy of intravenous immunoglobulins (IVIG) in this disorder remain unclear. We show that monocytes from patients with...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3006277

    authors: Sehgal K,Guo X,Koduru S,Shah A,Lin A,Yan X,Dhodapkar KM

    更新日期:2013-07-10 00:00:00

  • Controlled-release mitochondrial protonophore (CRMP) reverses dyslipidemia and hepatic steatosis in dysmetabolic nonhuman primates.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aay0284

    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

  • Thrombin contributes to cancer immune evasion via proteolysis of platelet-bound GARP to activate LTGF-β.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aay4860

    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

  • Hedgehog agonist therapy corrects structural and cognitive deficits in a Down syndrome mouse model.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3005983

    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

  • Dengue virus NS1 triggers endothelial permeability and vascular leak that is prevented by NS1 vaccination.

    abstract::The four dengue virus serotypes (DENV1 to DENV4) are mosquito-borne flaviviruses that cause up to ~100 million cases of dengue annually worldwide. Severe disease is thought to result from immunopathogenic processes involving serotype cross-reactive antibodies and T cells that together induce vasoactive cytokines, caus...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaa3787

    authors: Beatty PR,Puerta-Guardo H,Killingbeck SS,Glasner DR,Hopkins K,Harris E

    更新日期:2015-09-09 00:00:00

  • Staged induction of HIV-1 glycan-dependent broadly neutralizing antibodies.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aai7514

    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

  • Hyperpolarization-activated cyclic nucleotide-gated 2 (HCN2) ion channels drive pain in mouse models of diabetic neuropathy.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aam6072

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

    更新日期:2017-09-27 00:00:00

  • Remdesivir (GS-5734) protects African green monkeys from Nipah virus challenge.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aau9242

    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

  • Lysozyme elicits pain during nerve injury by neuronal Toll-like receptor 4 activation and has therapeutic potential in neuropathic pain.

    abstract::The role of neuronal Toll-like receptor 4 (TLR4) in nerve injury is being pursued actively. However, the endogenous activation of neuronal TLR4 during neuroinflammation, in absence of the participation of glial TLR4, remains elusive. Here, we identified lysozyme as an endogenous activator of neuronal TLR4 signaling du...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aav4176

    authors: Yadav S,Surolia A

    更新日期:2019-08-07 00:00:00

  • CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia.

    abstract::Adults with relapsed B cell acute lymphoblastic leukemia (B-ALL) have a dismal prognosis. Only those patients able to achieve a second remission with no minimal residual disease (MRD) have a hope for long-term survival in the context of a subsequent allogeneic hematopoietic stem cell transplantation (allo-HSCT). We ha...

    journal_title:Science translational medicine

    pub_type: 临床试验,杂志文章

    doi:10.1126/scitranslmed.3005930

    authors: Brentjens RJ,Davila ML,Riviere I,Park J,Wang X,Cowell LG,Bartido S,Stefanski J,Taylor C,Olszewska M,Borquez-Ojeda O,Qu J,Wasielewska T,He Q,Bernal Y,Rijo IV,Hedvat C,Kobos R,Curran K,Steinherz P,Jurcic J,Rosenbl

    更新日期:2013-03-20 00:00:00