Federal Agency Research Papers
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This section is for 'Therapeutic Discovery' category papers: these research articles report discoveries of new drug molecules or therapeutic approaches. These drugs are tested in experimental animal models, but not yet in humans.
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Dear medical scientists. We all know that not all public information is accessible information. If we don't act, people whom we ultimately serve (not our PIs!) such as patients and their families won't even know that these promising medical discoveries were ever published. The last thing we want as scientists is to have our research buried in libraries! Not only our organization makes the information easier to understand, we also make them easily searchable and freely accessible. Moreover, our endeavor is a really good way to demonstrate the importance of federal funding. Without NIH and NSF funding, these discoveries would have never come to light.
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Please help translate them into layman's term. Translated version will be posted here (http://www.opensciencedb.com/medical-research.html). Please sign up by writing your name and address next to the paper you'd like to summarize. And then use the form below to submit your summary! Feel free to add your own or others' research papers that meet this guideline (http://www.opensciencedb.com/how-it-works.html).
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USE THIS FORM TO SUBMIT YOUR SUMMARY! (CLICK)
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Title of the paperLinkYour name Your email addressStatus
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Loss and Gain of MeCP2 Cause Similar Hippocampal Circuit Dysfunction that Is Rescued by Deep Brain Stimulation in a Rett Syndrome Mouse Model.
https://www.ncbi.nlm.nih.gov/pubmed/27499081Julia Nussbacherjnussbac@ucsd.edu
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Forniceal deep brain stimulation rescues hippocampal memory in Rett syndrome mice.www.nature.com/articles/nature15694Katina Calakoskatina.calakos@yale.edu
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Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides.www.nature.com/articles/nature16159Mark Sabbaghmsabbag2@jhmi.educompleted
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Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiencyhttp://stm.sciencemag.org/content/8/335/335ra57
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CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous diseasehttp://stm.sciencemag.org/content/9/372/eaah3480Michael Pokrassmpokras1@jhmi.edu
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Neural Stem Cells Secreting Anti-HER2 Antibody Improve Survival in a Preclinical Model of HER2 Overexpressing Breast Cancer Brain Metastaseshttp://onlinelibrary.wiley.com/doi/10.1002/stem.2109/abstractDaniel Garciadag032@ucsd.educompleted
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Biologic-free mechanically induced muscle regenerationhttp://www.pnas.org/content/113/6/1534.abstract#corresp-1
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Silencing HoxA1 by Intraductal Injection of siRNA Lipidoid Nanoparticles Prevents Mammary Tumor Progression in Micehttp://stm.sciencemag.org/content/6/217/217ra2
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Shear-Activated Nanoparticle Aggregates Combined With Temporary Endovascular Bypass to Treat Large Vessel Occlusionhttp://stroke.ahajournals.org/content/early/2015/10/22/STROKEAHA.115.011063.abstract
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Injectable, spontaneously assembling, inorganic scaffolds modulate immune cells in vivo and increase vaccine efficacyhttp://www.nature.com/nbt/journal/v33/n1/full/nbt.3071.html#affil-authAna Milosevicamilosevic@rockefeller.edu
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Defining Hsp70 Subnetworks in Dengue Virus Replication Reveals Key Vulnerability in Flavivirus Infectionhttp://www.cell.com/cell/abstract/S0092-8674(15)01402-6
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A Therapeutic Antibody Targeting BACE1 Inhibits Amyloid-β Production in Vivohttp://stm.sciencemag.org/content/3/84/84ra43
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New IDH1 mutant inhibitors for treatment of acute myeloid leukemiahttp://www.nature.com/nchembio/journal/v11/n11/full/nchembio.1930.html#affil-authSam Stonersastoner@ucsd.edu
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Enzyme replacement with PEGylated cystathionine β-synthase ameliorates homocystinuria in murine modelhttp://www.jci.org/articles/view/85396
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Nanogel-based delivery of mycophenolic acid ameliorates systemic lupus erythematosus in mice.http://www.ncbi.nlm.nih.gov/pubmed/23454752
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Cutting Edge: Nanogel-Based Delivery of an Inhibitor of CaMK4 to CD4+ T Cells Suppresses Experimental Autoimmune Encephalomyelitis and Lupus-like Disease in Micehttp://www.jimmunol.org/content/195/12/5533.abstractDivij Mathewdivij.mathew@ucdenver.edu
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ENPP1-Fc prevents mortality and vascular calcifications in rodent model of generalized arterial calcification of infancyhttp://www.nature.com/ncomms/2015/151201/ncomms10006/full/ncomms10006.html
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BKM-120 (Buparlisib): A Phosphatidyl-Inositol-3 Kinase Inhibitor with Anti-Invasive Properties in Glioblastomahttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC4742861/
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Tofacitinib Citrate for the Treatment of Vitiligohttp://archderm.jamanetwork.com/article.aspx?articleid=2323633
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Killing two birds with one stone: oral tofacitinib reverses alopecia universalis in a patient with plaque psoriasis.http://www.ncbi.nlm.nih.gov/pubmed/24940651Samantha Joness7jones@ucsd.educompleted
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AZD9496: An Oral Estrogen Receptor Inhibitor That Blocks the Growth of ER-Positive and ESR1-Mutant Breast Tumors in Preclinical Modelshttp://cancerres.aacrjournals.org/content/early/2016/05/20/0008-5472.CAN-15-2357.abstractJordan Whitejwhit106@jhu.edu
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Renalase expression by melanoma and tumor associated-macrophages promotes tumor growth through a STAT3-mediated mechanismhttp://cancerres.aacrjournals.org/content/early/2016/05/05/0008-5472.CAN-15-1524.abstract?sid=675729fc-c54a-494f-956e-0ee786b55cb9Talia Henklethenkle1@jhmi.eduCompleted
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Chronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogshttp://www.jci.org/articles/view/41329Sarah Shiressshires@ucsd.edu
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Protection against malaria at 1 year and immune correlates following PfSPZ vaccinationhttp://www.nature.com/nm/journal/vaop/ncurrent/full/nm.4110.html#affil-authSamantha Jones
samantha.helen.jones@gmail.com
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Central injection of fibroblast growth factor 1 induces sustained remission of diabetic hyperglycemia in rodentshttp://www.nature.com/nm/journal/vaop/ncurrent/full/nm.4101.html#affil-auth
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Therapeutic bispecific T-cell engager antibody targeting the intracellular oncoprotein WT1http://www.nature.com/nbt/journal/v33/n10/full/nbt.3349.html#affil-authHeather Kinkeadhkinkea1@jhmi.eduCompleted
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An injectable nanoparticle generator enhances delivery of cancer therapeuticshttp://www.nature.com/nbt/journal/v34/n4/full/nbt.3506.html#affil-authHannah Vaughanhvaughan@jhmi.eduCompleted
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A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn micehttp://www.nature.com/nbt/journal/v34/n3/full/nbt.3469.html#affil-auth
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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivohttp://www.nature.com/nbt/journal/v34/n3/full/nbt.3471.html#affil-authMark Sabbaghmsabbag2@jhmi.educompleted
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Nanoparticle-formulated siRNA targeting integrins inhibits hepatocellular carcinoma progression in micehttp://www.nature.com/ncomms/2014/140521/ncomms4869/full/ncomms4869.html
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Biopolymer implants enhance the efficacy of adoptive T-cell therapyhttp://www.nature.com/nbt/journal/v33/n1/full/nbt.3104.html#affil-auth
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Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivohttp://www.nature.com/nbt/journal/v28/n8/full/nbt.1663.htmlSunandini Sridharssridhar@rockefeller.edu
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PP2A-activating drugs selectively eradicate TKI-resistant chronic myeloid leukemic stem cellshttp://www.jci.org/articles/view/68951
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Antibody-based targeting of FGFR3 in bladder carcinoma and t(4;14)-positive multiple myeloma in micehttp://www.jci.org/articles/view/38017
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Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteinshttp://www.nature.com/nm/journal/vaop/ncurrent/full/nm.4097.html#affil-authNina Gaonjgao@ucsd.eduIn preperation
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Early short-term treatment with neutralizing human monoclonal antibodies halts SHIV infection in infant macaqueshttp://www.nature.com/nm/journal/v22/n4/full/nm.4063.html#affil-authSamantha Joness7jones@ucsd.educompleted
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Long-term glycemic control using polymer-encapsulated human stem cell–derived beta cells in immune-competent micehttp://www.nature.com/nm/journal/v22/n3/full/nm.4030.html#affil-auth
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au-driven 26S proteasome impairment and cognitive dysfunction can be prevented early in disease by activating cAMP-PKA signalinghttp://www.nature.com/nm/journal/v22/n1/full/nm.4011.html
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Combined inhibition of BET family proteins and histone deacetylases as a potential epigenetics-based therapy for pancreatic ductal adenocarcinomahttp://www.nature.com/nm/journal/v21/n10/full/nm.3952.html#affil-auth
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PPAR-δ is repressed in Huntington's disease, is required for normal neuronal function and can be targeted therapeuticallyhttp://www.nature.com/nm/journal/v22/n1/full/nm.4003.html#affil-authLauren Gonzalezlauren.e.gonzalez@yale.edu
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Remote regulation of glucose homeostasis in mice using genetically encoded nanoparticleshttp://www.nature.com/nm/journal/v21/n1/full/nm.3730.html#affil-authAna Milosevicamilosevic@rockefeller.edu
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Perinatal choline supplementation improves cognitive functioning and emotion regulation in the Ts65Dn mouse model of Down syndrome.http://www.ncbi.nlm.nih.gov/pubmed/20528079?dopt=Abstract&holding=npg
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Mitochondrial iron chelation ameliorates cigarette smoke–induced bronchitis and emphysema in micehttp://www.nature.com/nm/journal/v22/n2/full/nm.4021.html#affil-auth
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Inhibition of mechanical allodynia in neuropathic pain by TLR5-mediated A-fiber blockadehttp://www.nature.com/nm/journal/v21/n11/full/nm.3978.html#affil-auth
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Targeting the MLL complex in castration-resistant prostate cancerhttp://www.nature.com/nm/journal/v21/n4/full/nm.3830.html#affil-auth
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An inhibitor of the protein kinases TBK1 and IKK-ε improves obesity-related metabolic dysfunctions in micehttp://www.nature.com/nm/journal/v19/n3/full/nm.3082.html#affil-auth
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Pharmacologic inhibition of histone demethylation as a therapy for pediatric brainstem gliomahttp://www.nature.com/nm/journal/v20/n12/full/nm.3716.html#affil-authAlex Miner
alexandra.miner@icahn.mssm.edu
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Dipeptidylpeptidase 4 negatively regulates colony-stimulating factor activity and stress hematopoiesishttp://www.nature.com/nm/journal/v18/n12/full/nm.2991.html#affil-auth
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Pharmacological targeting of actin-dependent dynamin oligomerization ameliorates chronic kidney disease in diverse animal modelshttp://www.nature.com/nm/journal/v21/n6/full/nm.3843.html#affil-authA. Erzbergeraerzberger@rockefeller.edu
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CSF-1R inhibition alters macrophage polarization and blocks glioma progressionhttp://www.nature.com/nm/journal/v19/n10/full/nm.3337.html#affil-authSuji Jeong
sujeong2017@u.northwestern.edu
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Alopecia areata is driven by cytotoxic T lymphocytes and is reversed by JAK inhibitionhttp://www.nature.com/nm/journal/v20/n9/full/nm.3645.html#affil-auth
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Effective and selective targeting of leukemia cells using a TORC1/2 kinase inhibitorhttp://www.nature.com/nm/journal/v16/n2/full/nm.2091.html#affil-auth
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A Gpr120-selective agonist improves insulin resistance and chronic inflammation in obese mice
http://www.nature.com/nm/journal/v20/n8/full/nm.3614.html#affil-auth
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Design of pathway preferential estrogens that provide beneficial metabolic and vascular effects without stimulating reproductive tissueshttp://stke.sciencemag.org/content/9/429/ra53.abstractJenelle Wallacejenellewallace@fas.harvard.edu
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Targeted fibrillar nanocarbon RNAi treatment of acute kidney injuryhttp://stm.sciencemag.org/content/8/331/331ra39
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A long-duration dihydroorotate dehydrogenase inhibitor (DSM265) for prevention and treatment of malariahttp://stm.sciencemag.org/content/7/296/296ra111
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Repurposing of the antihistamine chlorcyclizine and related compounds for treatment of hepatitis C virus infectionhttp://stm.sciencemag.org/content/7/282/282ra49Mac Critemac.crite@yale.educompleted
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Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosahttp://stm.sciencemag.org/content/6/264/264ra163Amy Vandiveraunterm1@jhmi.edu
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Treatment and Prevention of Urinary Tract Infection with Orally Active FimH Inhibitorshttp://stm.sciencemag.org/content/3/109/109ra115Saugat Poudelsapoudel@ucsd.edu
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Diamond-Lipid Hybrids Enhance Chemotherapeutic Tolerance and Mediate Tumor Regressionhttp://onlinelibrary.wiley.com/doi/10.1002/adma.201300343/abstract
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Therapy with CTLA4-Ig and an antiviral monoclonal antibody controls chikungunya virus arthritishttp://stm.sciencemag.org/content/9/375/eaah3438
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Tumor-homing cytotoxic human induced neural stem cells for cancer therapyhttp://stm.sciencemag.org/content/9/375/eaah6510
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Spinal Cord Stimulation Restores Locomotion in Animal Models of Parkinson's Diseasehttp://science.sciencemag.org/content/323/5921/1578Sarah Bakersbaker@rockefeller.educompleted
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Lentiviral Overexpression of GRK6 Alleviates l-Dopa–Induced Dyskinesia in Experimental Parkinson’s Diseasehttp://stm.sciencemag.org/content/2/28/28ra28
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Targeting ATM ameliorates mutant Huntingtin toxicity in cell and animal models of Huntington’s diseasehttp://stm.sciencemag.org/content/6/268/268ra178
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Eradication of large established tumors in mice by combination immunotherapy that engages innate and adaptive immune responseshttp://www.nature.com/nm/journal/v22/n12/full/nm.4200.html#affil-authHeather Kinkeadhkinkea1@jhmi.eduCompleted
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PIM1 kinase inhibition as a targeted therapy against triple-negative breast tumors with elevated MYC expressionhttp://www.nature.com/nm/journal/v22/n11/full/nm.4213.html#affil-auth
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A MYC–aurora kinase A protein complex represents an actionable drug target in p53-altered liver cancerhttp://www.nature.com/nm/journal/v22/n7/full/nm.4107.html
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Tau-driven 26S proteasome impairment and cognitive dysfunction can be prevented early in disease by activating cAMP-PKA signalinghttp://www.nature.com/nm/journal/v22/n1/full/nm.4011.html#affil-authEmily Grassoegrasso2@jhu.eduCompleted
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Fenretinide: A Novel Treatment for Endometrial Cancerhttp://journals.plos.org/plosone/article?id=10.1371/journal.pone.0110410
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Treatment of Prion Disease with Heterologous Prion Proteinshttp://journals.plos.org/plosone/article?id=10.1371/journal.pone.0131993
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Targeting Heat Shock Protein 90 for the Treatment of Malignant Pheochromocytomahttp://journals.plos.org/plosone/article?id=10.1371/journal.pone.0056083
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Preclinical Validation of a Single-Treatment Infusion Modality That Can Eradicate Extremity Melanomashttp://cancerres.aacrjournals.org/content/76/22/6620
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Efficacy of Adoptive T-cell Therapy Is Improved by Treatment with the Antioxidant N-Acetyl Cysteine, Which Limits Activation-Induced T-cell Deathhttp://cancerres.aacrjournals.org/content/76/20/6006
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HSP70 Inhibition Limits FAK-Dependent Invasion and Enhances the Response to Melanoma Treatment with BRAF Inhibitorshttp://cancerres.aacrjournals.org/content/76/9/2720
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Photodynamic Therapy Synergizes with Irinotecan to Overcome Compensatory Mechanisms and Improve Treatment Outcomes in Pancreatic Cancerhttp://cancerres.aacrjournals.org/content/76/5/1066
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MicroRNA-21 Lowers Blood Pressure in Spontaneous Hypertensive Rats by Upregulating Mitochondrial Translationhttp://circ.ahajournals.org/content/134/10/734Ethan Peretsethan.perets@yale.edu
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Overexpression of Tbx20 in Adult Cardiomyocytes Promotes Proliferation and Improves Cardiac Function After Myocardial Infarctionhttp://circ.ahajournals.org/content/133/11/1081
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Restoration of Impaired Endothelial Myocyte Enhancer Factor 2 Function Rescues Pulmonary Arterial Hypertensionhttp://circ.ahajournals.org/content/131/2/190
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Strategy for Identifying Repurposed Drugs for the Treatment of Cerebral Cavernous Malformationhttp://circ.ahajournals.org/content/131/3/289
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Double-Stranded RNA–Dependent Protein Kinase Deficiency Protects the Heart From Systolic Overload-Induced Congestive Heart Failurehttp://circ.ahajournals.org/content/129/13/1397
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Mammalian Target of Rapamycin Complex 2 (mTORC2) Coordinates Pulmonary Artery Smooth Muscle Cell Metabolism, Proliferation, and Survival in Pulmonary Arterial Hypertensionhttp://circ.ahajournals.org/content/129/8/864
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Transient rapamycin treatment can increase lifespan and healthspan in middle-aged micehttps://elifesciences.org/content/5/e16351
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Evolutionary dynamics of cancer in response to targeted combination therapyhttps://elifesciences.org/content/2/e00747Heather Kinkeadhkinkea1@jhmi.educompleted
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Cyclooxygenase-2 inhibition reduces stress-induced affective pathologyhttps://elifesciences.org/content/5/e14137Ana Milosevicamilosevic@rockefelelr.edu
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Acute inhibition of neurosteroid estrogen synthesis suppresses status epilepticus in an animal modelhttps://elifesciences.org/content/5/e12917
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Oxytocin improves behavioral and electrophysiological deficits in a novel Shank3-deficient rathttps://elifesciences.org/content/6/e18904
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A CNS-permeable Hsp90 inhibitor rescues synaptic dysfunction and memory loss in APP-overexpressing Alzheimer’s mouse model via an HSF1-mediated mechanismhttp://www.nature.com/mp/journal/vaop/ncurrent/full/mp2016104a.html
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Serotonin 2C receptor antagonists induce fast-onset antidepressant effectshttp://www.nature.com/mp/journal/v19/n10/full/mp2013144a.htmlDavid Ottenheimerdavid.ottenheimer@jhu.educompleted
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Preventive and therapeutic effects of Smad7 on radiation-induced oral mucositishttp://www.nature.com/nm/journal/v19/n4/full/nm.3118.html#affil-auth
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