Amphiphysin 2 modulation rescues myotubular myopathy and prevents focal adhesion defects in mice.

abstract：:Pathogenic immunoglobulin G (IgG) autoantibodies characterize some human autoimmune diseases; their high concentration and long half-life are dependent on recycling by the neonatal Fc receptor (FcRn). Inhibition of FcRn is an attractive new treatment concept for IgG-mediated autoimmune diseases. Rozanolixizumab (UCB76...

journal_title：Science translational medicine

authors： Kiessling P,Lledo-Garcia R,Watanabe S,Langdon G,Tran D,Bari M,Christodoulou L,Jones E,Price G,Smith B,Brennan F,White I,Jolles S

更新日期：2017-11-01 00:00:00

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

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

abstract：:Imaging, including its use for innovative tissue sampling, is slowly being recognized as playing a pivotal role in drug development, clinical trial design, and more effective delivery and monitoring of molecular therapies. The challenge is that, while a considerable number of new imaging technologies and new targeted ...

journal_title：Science translational medicine

authors： Weissleder R,Schwaiger MC,Gambhir SS,Hricak H

更新日期：2016-09-07 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：: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：: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

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

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

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

abstract：:Acute graft-versus-host disease (aGVHD) results from a robust response of donor T cells transferred during hematopoietic stem cell transplantation (HSCT) to allogeneic peptide-major histocompatibility complex antigens. Previous investigations have not identified T cell subsets that selectively mediate either protectiv...

journal_title：Science translational medicine

authors： Morris GP,Uy GL,Donermeyer D,Dipersio JF,Allen PM

更新日期：2013-06-05 00:00:00

abstract：:Three-dimensional (3D) titanium-mesh scaffolds offer many advantages over autologous bone grafting for the regeneration of challenging large segmental bone defects. Our study supports the hypothesis that endogenous bone defect regeneration can be promoted by mechanobiologically optimized Ti-mesh scaffolds. Using finit...

journal_title：Science translational medicine

authors： Pobloth AM,Checa S,Razi H,Petersen A,Weaver JC,Schmidt-Bleek K,Windolf M,Tatai AÁ,Roth CP,Schaser KD,Duda GN,Schwabe P

更新日期：2018-01-10 00:00:00

abstract：:Troublesome involuntary movements in the absence of dopaminergic medication, so-called off-medication dyskinesias, are a serious adverse effect of fetal neural grafts that hinders the development of cell-based therapies for Parkinson's disease. The mechanisms underlying these dyskinesias are not well understood, and i...

journal_title：Science translational medicine

authors： Politis M,Wu K,Loane C,Quinn NP,Brooks DJ,Rehncrona S,Bjorklund A,Lindvall O,Piccini P

更新日期：2010-06-30 00:00:00

abstract：:Despite the remarkable enhancements in breast cancer classification and characterization, an exhaustive comprehension of the underlying carcinogenic processes and an accurate prediction of its clinical behavior are yet to be achieved. On the wave of recent scientific advances and clinical findings, new cutting-edge te...

journal_title：Science translational medicine

authors： Reis-Filho JS,Weigelt B,Fumagalli D,Sotiriou C

更新日期：2010-09-01 00:00:00

abstract：:In severe spinal cord injuries, the tracts conveying motor commands to the spinal cord are disrupted, resulting in paralysis, but many patients still have small numbers of spared fibers. We have found that excitatory deep brain stimulation (DBS) of the mesencephalic locomotor region (MLR), an important control center ...

journal_title：Science translational medicine

authors： Bachmann LC,Matis A,Lindau NT,Felder P,Gullo M,Schwab ME

更新日期：2013-10-23 00:00:00

abstract：:The recent Perspective entitled "Power of rare diseases: Found in translation" undervalues the contributions of academic research in first-in-human studies. ...

journal_title：Science translational medicine

authors： van der Meer JW,Simon A,Dinarello CA

更新日期：2014-01-15 00:00:00

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

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

abstract：:KRAS represents an excellent therapeutic target in lung cancer, the most commonly mutated form of which can now be blocked using KRAS-G12C mutant-specific inhibitory trial drugs. Lung adenocarcinoma cells harboring KRAS mutations have been shown previously to be selectively sensitive to inhibition of mitogen-activated...

journal_title：Science translational medicine

authors： Molina-Arcas M,Moore C,Rana S,van Maldegem F,Mugarza E,Romero-Clavijo P,Herbert E,Horswell S,Li LS,Janes MR,Hancock DC,Downward J

更新日期：2019-09-18 00:00:00

abstract：:Because of the well-established therapeutic benefit of boosting antitumor responses through blockade of the T cell inhibitory receptor PD-1, it has been proposed that PD-1 blockade could also be useful in infectious disease settings, including Mycobacterium tuberculosis (Mtb) infection. However, in preclinical models,...

journal_title：Science translational medicine

authors： Barber DL,Sakai S,Kudchadkar RR,Fling SP,Day TA,Vergara JA,Ashkin D,Cheng JH,Lundgren LM,Raabe VN,Kraft CS,Nieva JJ,Cheever MA,Nghiem PT,Sharon E

更新日期：2019-01-16 00:00:00

abstract：:Long-acting GSK1265744 (GSK744 LA) is a strand transfer inhibitor of the HIV/SIV (simian immunodeficiency virus) integrase and was shown to be an effective preexposure prophylaxis (PrEP) agent in a low-dose intrarectal SHIV (simian-human immunodeficiency virus) rhesus macaque challenge model. We examined the pharmacok...

journal_title：Science translational medicine

authors： Andrews CD,Yueh YL,Spreen WR,St Bernard L,Boente-Carrera M,Rodriguez K,Gettie A,Russell-Lodrigue K,Blanchard J,Ford S,Mohri H,Cheng-Mayer C,Hong Z,Ho DD,Markowitz M

更新日期：2015-01-14 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：:Rapid advances in the forward engineering of genetic circuitry in living cells has positioned synthetic biology as a potential means to solve numerous biomedical problems, including disease diagnosis and therapy. One challenge in exploiting synthetic biology for translational applications is to engineer microbes that ...

journal_title：Science translational medicine

authors： Danino T,Prindle A,Kwong GA,Skalak M,Li H,Allen K,Hasty J,Bhatia SN

更新日期：2015-05-27 00:00:00

abstract：:Regulatory T cells (T(regs)) manipulated ex vivo have potential as cellular therapeutics in autoimmunity and transplantation. Although it is possible to expand naturally occurring T(regs), an attractive alternative possibility, particularly suited to solid organ and bone marrow transplantation, is the stimulation of t...

journal_title：Science translational medicine

authors： Feng G,Nadig SN,Bäckdahl L,Beck S,Francis RS,Schiopu A,Whatcott A,Wood KJ,Bushell A

更新日期：2011-05-18 00:00:00

abstract：:Treatment of life-threatening Epstein-Barr virus (EBV)-associated tumors remains a great challenge, especially for patients with relapsed or refractory disease. Here, we found that exosomes derived from phosphoantigen-expanded Vδ2-T cells (Vδ2-T-Exos) contained death-inducing ligands (FasL and TRAIL), an activating re...

journal_title：Science translational medicine

authors： Wang X,Xiang Z,Liu Y,Huang C,Pei Y,Wang X,Zhi H,Wong WH,Wei H,Ng IO,Lee PP,Chan GC,Lau YL,Tu W

更新日期：2020-09-30 00:00:00

abstract：:Inherited retinal degenerative diseases, a genetically and phenotypically heterogeneous group of disorders, affect the function of photoreceptor cells and are among the leading causes of blindness. Recent advances in molecular genetics and cell biology are elucidating the pathophysiological mechanisms underlying these...

journal_title：Science translational medicine

authors： Scholl HP,Strauss RW,Singh MS,Dalkara D,Roska B,Picaud S,Sahel JA

更新日期：2016-12-07 00:00:00

abstract：:Evaluating individual research performance is a complex task that ideally examines productivity, scientific impact, and research quality--a task that metrics alone have been unable to achieve. In January 2011, the French Academy of Sciences published a report on current bibliometric (citation metric) methods for evalu...

journal_title：Science translational medicine

authors： Sahel JA

更新日期：2011-05-25 00:00:00

abstract：:Cerebral cavernous malformation (CCM) is a genetic, cerebrovascular disease. Familial CCM is caused by genetic mutations in KRIT1, CCM2, or PDCD10 Disease onset is earlier and more severe in individuals with PDCD10 mutations. Recent studies have shown that lesions arise from excess mitogen-activated protein kinase kin...

journal_title：Science translational medicine

authors： Tang AT,Sullivan KR,Hong CC,Goddard LM,Mahadevan A,Ren A,Pardo H,Peiper A,Griffin E,Tanes C,Mattei LM,Yang J,Li L,Mericko-Ishizuka P,Shen L,Hobson N,Girard R,Lightle R,Moore T,Shenkar R,Polster SP,Roedel CJ,Li

更新日期：2019-11-27 00:00:00

abstract：:Public health emergencies, such as an Ebola disease outbreak, provide a complex and challenging environment for the evaluation of candidate vaccines. Here, we outline the need for flexible and responsive vaccine trial designs to be used in public health emergencies, and we summarize recommendations for their use in th...

journal_title：Science translational medicine

authors： Dean NE,Gsell PS,Brookmeyer R,De Gruttola V,Donnelly CA,Halloran ME,Jasseh M,Nason M,Riveros X,Watson CH,Henao-Restrepo AM,Longini IM

更新日期：2019-07-03 00:00:00

abstract：:The development of new approaches to cholera control relies on an accurate understanding of cholera epidemiology. However, most information on cholera incidence lacks laboratory confirmation and instead relies on surveillance systems reporting medically attended acute watery diarrhea. If recent infections could be ide...

journal_title：Science translational medicine

authors： Azman AS,Lessler J,Luquero FJ,Bhuiyan TR,Khan AI,Chowdhury F,Kabir A,Gurwith M,Weil AA,Harris JB,Calderwood SB,Ryan ET,Qadri F,Leung DT

更新日期：2019-02-20 00:00:00

abstract：:In acute and chronic inflammation, neutrophils and platelets, both of which promote monocyte recruitment, are often activated simultaneously. We investigated how secretory products of neutrophils and platelets synergize to enhance the recruitment of monocytes. We found that neutrophil-borne human neutrophil peptide 1 ...

journal_title：Science translational medicine

authors： Alard JE,Ortega-Gomez A,Wichapong K,Bongiovanni D,Horckmans M,Megens RT,Leoni G,Ferraro B,Rossaint J,Paulin N,Ng J,Ippel H,Suylen D,Hinkel R,Blanchet X,Gaillard F,D'Amico M,von Hundelshausen P,Zarbock A,Scheiermann

更新日期：2015-12-09 00:00:00

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

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

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

authors： O'Neill LA

更新日期：2010-06-16 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