Beyond disease, how biomedical engineering can improve global health.

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：:Inhaled drugs offer advantages, such as rapid onset of action, but require formulations and delivery systems that reproducibly and conveniently administer the drug. CVT-301 is a powder formulation of levodopa delivered by a breath-actuated inhaler that has been developed for treating OFF episodes (motor fluctuations b...

journal_title：Science translational medicine

authors： Lipp MM,Batycky R,Moore J,Leinonen M,Freed MI

更新日期：2016-10-12 00:00:00

abstract：:Disease-free infection in HIV-infected adults is associated with human leukocyte antigen-mediated suppression of viremia, whereas in the sooty mangabey and other healthy natural hosts of simian immunodeficiency virus (SIV), viral replication continues unabated. To better understand factors preventing HIV disease, we i...

journal_title：Science translational medicine

authors： Muenchhoff M,Adland E,Karimanzira O,Crowther C,Pace M,Csala A,Leitman E,Moonsamy A,McGregor C,Hurst J,Groll A,Mori M,Sinmyee S,Thobakgale C,Tudor-Williams G,Prendergast AJ,Kloverpris H,Roider J,Leslie A,Shingadia D

更新日期：2016-09-28 00:00:00

abstract：:Centronuclear myopathies (CNMs) are severe diseases characterized by muscle weakness and myofiber atrophy. Currently, there are no approved treatments for these disorders. Mutations in the phosphoinositide 3-phosphatase myotubularin (MTM1) are responsible for X-linked CNM (XLCNM), also called myotubular myopathy, wher...

journal_title：Science translational medicine

authors： Lionello VM,Nicot AS,Sartori M,Kretz C,Kessler P,Buono S,Djerroud S,Messaddeq N,Koebel P,Prokic I,Hérault Y,Romero NB,Laporte J,Cowling BS

更新日期：2019-03-20 00:00:00

abstract：:Sporadic inclusion body myositis (sIBM) is the commonest severe myopathy in patients more than 50 years of age. Previous therapeutic trials have targeted the inflammatory features of sIBM but all have failed. Because protein dyshomeostasis may also play a role in sIBM, we tested the effects of targeting this feature o...

journal_title：Science translational medicine

authors： Ahmed M,Machado PM,Miller A,Spicer C,Herbelin L,He J,Noel J,Wang Y,McVey AL,Pasnoor M,Gallagher P,Statland J,Lu CH,Kalmar B,Brady S,Sethi H,Samandouras G,Parton M,Holton JL,Weston A,Collinson L,Taylor JP,Schia

更新日期：2016-03-23 00:00:00

abstract：:Children who survive preterm birth exhibit persistent unexplained disturbances in cerebral cortical growth with associated cognitive and learning disabilities. The mechanisms underlying these deficits remain elusive. We used ex vivo diffusion magnetic resonance imaging to demonstrate in a preterm large-animal model th...

journal_title：Science translational medicine

authors： Dean JM,McClendon E,Hansen K,Azimi-Zonooz A,Chen K,Riddle A,Gong X,Sharifnia E,Hagen M,Ahmad T,Leigland LA,Hohimer AR,Kroenke CD,Back SA

更新日期：2013-01-16 00:00:00

abstract：:Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) anion channel. The most common CF-associated mutation is ΔF508, which deletes a phenylalanine in position 508. In vitro studies indicate that the resultant prot...

journal_title：Science translational medicine

authors： Ostedgaard LS,Meyerholz DK,Chen JH,Pezzulo AA,Karp PH,Rokhlina T,Ernst SE,Hanfland RA,Reznikov LR,Ludwig PS,Rogan MP,Davis GJ,Dohrn CL,Wohlford-Lenane C,Taft PJ,Rector MV,Hornick E,Nassar BS,Samuel M,Zhang Y,Richt

更新日期：2011-03-16 00:00:00

abstract：:Cooperative oncogenic effects resulting from the loss of PTEN and overexpression of MYC overcome the deleterious effects of endoplasmic reticulum stress not only to promote the growth of aggressive prostate cancer but also to expose a new therapy target for this disease (Nguyen et al, this issue). ...

journal_title：Science translational medicine

authors： Logothetis C,Aparicio A,Thompson TC

更新日期：2018-05-02 00:00:00

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

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

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

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

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

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

abstract：:Elevated serum levels of both total and allergen-specific immunoglobulin E (IgE) correlate with atopic diseases such as allergic rhinitis and allergic asthma. Neutralization of IgE by anti-IgE antibodies can effectively treat allergic asthma. Preclinical studies indicate that targeting membrane IgE-positive cells with...

journal_title：Science translational medicine

authors： Gauvreau GM,Harris JM,Boulet LP,Scheerens H,Fitzgerald JM,Putnam WS,Cockcroft DW,Davis BE,Leigh R,Zheng Y,Dahlén B,Wang Y,Maciuca R,Mayers I,Liao XC,Wu LC,Matthews JG,O'Byrne PM

更新日期：2014-07-02 00:00:00

abstract：:New therapeutic approaches are urgently needed to improve survival outcomes for patients with necrotizing pneumonia caused by Staphylococcus aureus One such approach is adjunctive treatment with intravenous immunoglobulin (IVIG), but clinical practice guidelines offer conflicting recommendations. In a preclinical rabb...

journal_title：Science translational medicine

authors： Diep BA,Le VT,Badiou C,Le HN,Pinheiro MG,Duong AH,Wang X,Dip EC,Aguiar-Alves F,Basuino L,Marbach H,Mai TT,Sarda MN,Kajikawa O,Matute-Bello G,Tkaczyk C,Rasigade JP,Sellman BR,Chambers HF,Lina G

更新日期：2016-09-21 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：:Prenatal and postnatal treatment with a CFTR modifier attenuates pathological changes in a ferret model of cystic fibrosis (Sun et al., this issue). ...

journal_title：Science translational medicine

authors： Ferkol TW

更新日期：2019-03-27 00:00:00

abstract：:The epidermal growth factor receptor (EGFR) plays an important role in tissue homeostasis and tumor progression. However, cancer patients treated with EGFR inhibitors (EGFRIs) frequently develop acneiform skin toxicities, which are a strong predictor of a patient's treatment response. We show that the early inflammato...

journal_title：Science translational medicine

authors： Lichtenberger BM,Gerber PA,Holcmann M,Buhren BA,Amberg N,Smolle V,Schrumpf H,Boelke E,Ansari P,Mackenzie C,Wollenberg A,Kislat A,Fischer JW,Röck K,Harder J,Schröder JM,Homey B,Sibilia M

更新日期：2013-08-21 00:00:00

abstract：:Whole-genome sequencing of patient DNA can facilitate diagnosis of a disease, but its potential for guiding treatment has been under-realized. We interrogated the complete genome sequences of a 14-year-old fraternal twin pair diagnosed with dopa (3,4-dihydroxyphenylalanine)-responsive dystonia (DRD; Mendelian Inherita...

journal_title：Science translational medicine

authors： Bainbridge MN,Wiszniewski W,Murdock DR,Friedman J,Gonzaga-Jauregui C,Newsham I,Reid JG,Fink JK,Morgan MB,Gingras MC,Muzny DM,Hoang LD,Yousaf S,Lupski JR,Gibbs RA

更新日期：2011-06-15 00:00:00

abstract：:A study in this issue of Science Translational Medicine provides fresh insight into the role of autophagy in cancer. Kon et al. show that a specific type of autophagy, called chaperone-mediated autophagy (CMA), is consistently elevated in tumors and is required for optimal tumor growth and metastasis. Selective inhibi...

journal_title：Science translational medicine

authors： Thorburn A,Debnath J

更新日期：2011-11-16 00:00:00

abstract：:Studies have suggested a role for the mammalian (or mechanistic) target of rapamycin (mTOR) in skeletal development and homeostasis, yet there is no evidence connecting mTOR with the key signaling pathways that regulate skeletogenesis. We identified a parathyroid hormone (PTH)/PTH-related peptide (PTHrP)-salt-inducibl...

journal_title：Science translational medicine

authors： Csukasi F,Duran I,Barad M,Barta T,Gudernova I,Trantirek L,Martin JH,Kuo CY,Woods J,Lee H,Cohn DH,Krejci P,Krakow D

更新日期：2018-09-19 00:00:00

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

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

abstract：:Heparin-based anticoagulant drugs have been widely used for the prevention of blood clotting during surgical procedures and for the treatment of thromboembolic events. However, bleeding risks associated with these anticoagulants demand continuous monitoring and neutralization with suitable antidotes. Protamine, the on...

journal_title：Science translational medicine

authors： Shenoi RA,Kalathottukaren MT,Travers RJ,Lai BF,Creagh AL,Lange D,Yu K,Weinhart M,Chew BH,Du C,Brooks DE,Carter CJ,Morrissey JH,Haynes CA,Kizhakkedathu JN

更新日期：2014-10-29 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：: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 researc...

journal_title：Science translational medicine

authors： Connor E,Lombardi D,van den Anker J

更新日期：2009-10-14 00:00:00

abstract：:Recent studies have established the involvement of the fat mass and obesity-associated gene (FTO) in metabolic disorders such as obesity and diabetes. However, the precise molecular mechanism by which FTO regulates metabolism remains unknown. Here, we used a structure-based virtual screening of U.S. Food and Drug Admi...

journal_title：Science translational medicine

authors： Peng S,Xiao W,Ju D,Sun B,Hou N,Liu Q,Wang Y,Zhao H,Gao C,Zhang S,Cao R,Li P,Huang H,Ma Y,Wang Y,Lai W,Ma Z,Zhang W,Huang S,Wang H,Zhang Z,Zhao L,Cai T,Zhao YL,Wang F,Nie Y,Zhi G,Yang YG,Zhang EE,

更新日期：2019-04-17 00:00:00

abstract：:The research and development of innovative new therapies are expensive and risky processes, and companies require substantial resources if they are to translate basic science into modern biomedical products. When resources are limited, as in the current economic climate, biotechnology start-up companies are often amon...

journal_title：Science translational medicine

authors： Greenwood JC

更新日期：2010-01-06 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：:The August 2011 Clinical and Translational Science Awards conference "Using IT to Improve Community Health: How Health Care Reform Supports Innovation" convened four "Think Tank" sessions. Thirty individuals, representing various perspectives on community engagement, attended the "Health information technology (HIT) a...

journal_title：Science translational medicine

authors： Marriott LK,Nelson DA,Allen S,Calhoun K,Eldredge CE,Kimminau KS,Lucero RJ,Pineda-Reyes F,Rumala BB,Varanasi AP,Wasser JS,Shannon J

更新日期：2012-02-01 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：: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：:Hydrocephalus is a common and costly neurological condition caused by the overproduction and/or impaired resorption of cerebrospinal fluid (CSF). The current standard of care, ventricular catheters (shunts), is prone to failure, which can result in nonspecific symptoms such as headaches, dizziness, and nausea. Current...

journal_title：Science translational medicine

authors： Krishnan SR,Ray TR,Ayer AB,Ma Y,Gutruf P,Lee K,Lee JY,Wei C,Feng X,Ng B,Abecassis ZA,Murthy N,Stankiewicz I,Freudman J,Stillman J,Kim N,Young G,Goudeseune C,Ciraldo J,Tate M,Huang Y,Potts M,Rogers JA

更新日期：2018-10-31 00:00:00