More than baby steps: perspectives on pediatric translational research.

Abstract:

:Although children represent 25% of the U.S. population and nearly 40% of the world's population, pediatric product development typically lags behind that targeted at adults. Here the authors describe the challenges and opportunities of pediatric product development and the unique issues that surround pediatric research. Assuring advancement in pediatric health through clinical and translational medicine is a high priority and an investment in the future.

journal_name

Sci Transl Med

authors

Connor E,Lombardi D,van den Anker J

doi

10.1126/scitranslmed.3000152

subject

Has Abstract

pub_date

2009-10-14 00:00:00

pages

2cm2

issue

2

eissn

1946-6234

issn

1946-6242

pii

1/2/2cm2

journal_volume

1

pub_type

杂志文章
  • 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

  • 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

  • Long noncoding RNA Chast promotes cardiac remodeling.

    abstract::Recent studies highlighted long noncoding RNAs (lncRNAs) to play an important role in cardiac development. However, understanding of lncRNAs in cardiac diseases is still limited. Global lncRNA expression profiling indicated that several lncRNA transcripts are deregulated during pressure overload-induced cardiac hypert...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaf1475

    authors: Viereck J,Kumarswamy R,Foinquinos A,Xiao K,Avramopoulos P,Kunz M,Dittrich M,Maetzig T,Zimmer K,Remke J,Just A,Fendrich J,Scherf K,Bolesani E,Schambach A,Weidemann F,Zweigerdt R,de Windt LJ,Engelhardt S,Dandekar T,

    更新日期:2016-02-17 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

  • Achieving a nationwide learning health system.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3001456

    authors: Friedman CP,Wong AK,Blumenthal D

    更新日期:2010-11-10 00:00:00

  • Tissue-specific regulation of p53 by PKM2 is redox dependent and provides a therapeutic target for anthracycline-induced cardiotoxicity.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aau8866

    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

  • Off-target lapatinib activity sensitizes colon cancer cells through TRAIL death receptor up-regulation.

    abstract::Lapatinib, a dual HER2/EGFR (human epidermal growth factor receptor 2/epidermal growth factor receptor) inhibitor, is a recently approved targeted therapy for metastatic breast cancer. Because lapatinib enhances the efficacy of the chemotherapeutic agent capecitabine in breast cancer patients, we tested whether lapati...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3001384

    authors: Dolloff NG,Mayes PA,Hart LS,Dicker DT,Humphreys R,El-Deiry WS

    更新日期:2011-06-08 00:00:00

  • The inflammasome in atherosclerosis and type 2 diabetes.

    abstract::Atherosclerosis is the cause of morbiditiy for 70% of patients with type 2 diabetes. In both of these diseases, a protein complex known as the inflammasome is stimulated to activate interleukin-1β (IL-1β) and IL-18, which are pathogenic inflammatory cytokines. Triggers for the inflammasome are obesity-related factors,...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3001902

    authors: Masters SL,Latz E,O'Neill LA

    更新日期:2011-05-04 00:00:00

  • How to handle an industry in disruption: Intervene or laissez-faire?

    abstract::An analysis of four regions in four countries suggests that the best way to mitigate disruption of the pharmaceutical industry is not to ignore or stem it but to sponsor initiatives that keep scientists engaged. ...

    journal_title:Science translational medicine

    pub_type: 杂志文章,评审

    doi:10.1126/scitranslmed.aaa3679

    authors: Munos B

    更新日期:2015-05-06 00:00:00

  • Anchoring of intratumorally administered cytokines to collagen safely potentiates systemic cancer immunotherapy.

    abstract::The clinical application of cytokine therapies for cancer treatment remains limited due to severe adverse reactions and insufficient therapeutic effects. Although cytokine localization by intratumoral administration could address both issues, the rapid escape of soluble cytokines from the tumor invariably subverts thi...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaw2614

    authors: Momin N,Mehta NK,Bennett NR,Ma L,Palmeri JR,Chinn MM,Lutz EA,Kang B,Irvine DJ,Spranger S,Wittrup KD

    更新日期:2019-06-26 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

  • PTEN status determines chemosensitivity to proteasome inhibition in cholangiocarcinoma.

    abstract::Patient-derived xenografts (PDXs) and PDX-derived cells (PDCs) are useful in preclinical research. We performed a drug screening assay using PDCs and identified proteasome inhibitors as promising drugs for cholangiocarcinoma (CCA) treatment. Furthermore, we determined that phosphate and tensin homology deleted on chro...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aay0152

    authors: Jiang TY,Pan YF,Wan ZH,Lin YK,Zhu B,Yuan ZG,Ma YH,Shi YY,Zeng TM,Dong LW,Tan YX,Wang HY

    更新日期:2020-09-23 00:00:00

  • Endomicroscope sheds light on cervical tissue.

    abstract::A pilot study of a handheld fluorescence endomicroscope demonstrates both in vivo imaging and differentiation of normal from precancerous cervical tissue. ...

    journal_title:Science translational medicine

    pub_type: 评论,杂志文章

    doi:10.1126/scitranslmed.aal4989

    authors: Evans CL

    更新日期:2017-01-11 00:00:00

  • Molecular imaging of bacterial infections: Overcoming the barriers to clinical translation.

    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

    pub_type: 杂志文章,评审

    doi:10.1126/scitranslmed.aax8251

    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

  • Therapeutically relevant engraftment of a CRISPR-Cas9-edited HSC-enriched population with HbF reactivation in nonhuman primates.

    abstract::Reactivation of fetal hemoglobin (HbF) is being pursued as a treatment strategy for hemoglobinopathies. Here, we evaluated the therapeutic potential of hematopoietic stem and progenitor cells (HSPCs) edited with the CRISPR-Cas9 nuclease platform to recapitulate naturally occurring mutations identified in individuals w...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaw3768

    authors: Humbert O,Radtke S,Samuelson C,Carrillo RR,Perez AM,Reddy SS,Lux C,Pattabhi S,Schefter LE,Negre O,Lee CM,Bao G,Adair JE,Peterson CW,Rawlings DJ,Scharenberg AM,Kiem HP

    更新日期:2019-07-31 00:00:00

  • Intraindividual genome expression analysis reveals a specific molecular signature of psoriasis and eczema.

    abstract::Previous attempts to gain insight into the pathogenesis of psoriasis and eczema by comparing their molecular signatures were hampered by the high interindividual variability of those complex diseases. In patients affected by both psoriasis and nonatopic or atopic eczema simultaneously (n = 24), an intraindividual comp...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3008946

    authors: Quaranta M,Knapp B,Garzorz N,Mattii M,Pullabhatla V,Pennino D,Andres C,Traidl-Hoffmann C,Cavani A,Theis FJ,Ring J,Schmidt-Weber CB,Eyerich S,Eyerich K

    更新日期:2014-07-09 00:00:00

  • 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_title:Science translational medicine

    pub_type: 杂志文章,评审

    doi:10.1126/scitranslmed.aaq0810

    authors: Michael NL

    更新日期:2017-12-06 00:00:00

  • Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.abb5413

    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

  • CRISPR-enhanced engineering of therapy-sensitive cancer cells for self-targeting of primary and metastatic tumors.

    abstract::Tumor cells engineered to express therapeutic agents have shown promise to treat cancer. However, their potential to target cell surface receptors specific to the tumor site and their posttreatment fate have not been explored. We created therapeutic tumor cells expressing ligands specific to primary and recurrent tumo...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aao3240

    authors: Reinshagen C,Bhere D,Choi SH,Hutten S,Nesterenko I,Wakimoto H,Le Roux E,Rizvi A,Du W,Minicucci C,Shah K

    更新日期:2018-07-11 00:00:00

  • A vitamin to D-crease sunburn.

    abstract::High doses of oral vitamin D3 attenuate skin inflammation following experimentally induced sun damage. ...

    journal_title:Science translational medicine

    pub_type: 评论,杂志文章

    doi:10.1126/scitranslmed.aan5233

    authors: Scharschmidt TC

    更新日期:2017-06-14 00:00:00

  • Up-regulation of PD-L1, IDO, and T(regs) in the melanoma tumor microenvironment is driven by CD8(+) T cells.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3006504

    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

  • Development of personalized tumor biomarkers using massively parallel sequencing.

    abstract::Clinical management of human cancer is dependent on the accurate monitoring of residual and recurrent tumors. The evaluation of patient-specific translocations in leukemias and lymphomas has revolutionized diagnostics for these diseases. We have developed a method, called personalized analysis of rearranged ends (PARE...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3000702

    authors: Leary RJ,Kinde I,Diehl F,Schmidt K,Clouser C,Duncan C,Antipova A,Lee C,McKernan K,De La Vega FM,Kinzler KW,Vogelstein B,Diaz LA Jr,Velculescu VE

    更新日期:2010-02-24 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

  • Sensitization to immune checkpoint blockade through activation of a STAT1/NK axis in the tumor microenvironment.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aav7816

    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

  • Age-related intimal stiffening enhances endothelial permeability and leukocyte transmigration.

    abstract::Age is the most significant risk factor for atherosclerosis; however, the link between age and atherosclerosis is poorly understood. During both aging and atherosclerosis progression, the blood vessel wall stiffens owing to alterations in the extracellular matrix. Using in vitro and ex vivo models of vessel wall stiff...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3002761

    authors: Huynh J,Nishimura N,Rana K,Peloquin JM,Califano JP,Montague CR,King MR,Schaffer CB,Reinhart-King CA

    更新日期:2011-12-07 00:00:00

  • Vitamin C puts the pedal to the metal.

    abstract::Increased iron in cancer cells drives selective sensitization of tumors to ascorbate treatment to prolong survival. ...

    journal_title:Science translational medicine

    pub_type: 评论,杂志文章

    doi:10.1126/scitranslmed.aan2778

    authors: Venere M

    更新日期:2017-04-12 00:00:00

  • Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.

    abstract::Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease caused by loss of activity of α-l-iduronidase and attendant accumulation of the glycosaminoglycans dermatan sulfate and heparan sulfate. Current treatments are suboptimal and do not address residual disease including corneal clouding, skeletal deformi...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3001380

    authors: Dierenfeld AD,McEntee MF,Vogler CA,Vite CH,Chen AH,Passage M,Le S,Shah S,Jens JK,Snella EM,Kline KL,Parkes JD,Ware WA,Moran LE,Fales-Williams AJ,Wengert JA,Whitley RD,Betts DM,Boal AM,Riedesel EA,Gross W,Ellinwo

    更新日期:2010-12-01 00:00:00

  • Integration of early physiological responses predicts later illness severity in preterm infants.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.3001304

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

    更新日期:2010-09-08 00:00:00

  • High-throughput metabolomic analysis predicts mode of action of uncharacterized antimicrobial compounds.

    abstract::Rapidly spreading antibiotic resistance and the low discovery rate of new antimicrobial compounds demand more effective strategies for early drug discovery. One bottleneck in the drug discovery pipeline is the identification of the modes of action (MoAs) of new compounds. We have developed a rapid systematic metabolom...

    journal_title:Science translational medicine

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aal3973

    authors: Zampieri M,Szappanos B,Buchieri MV,Trauner A,Piazza I,Picotti P,Gagneux S,Borrell S,Gicquel B,Lelievre J,Papp B,Sauer U

    更新日期:2018-02-21 00:00:00

  • GLP-1 receptor agonists synergize with DYRK1A inhibitors to potentiate functional human β cell regeneration.

    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

    pub_type: 杂志文章

    doi:10.1126/scitranslmed.aaw9996

    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