Search Results - "Gollob, Jared"
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Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
Published in The Lancet (British edition) (04-01-2014)“…Summary Background Proprotein convertase subtilisin/kexin type 9 (PCSK9) binds to LDL receptors, leading to their degradation. Genetics studies have shown that…”
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Safety profile of RNAi nanomedicines
Published in Advanced drug delivery reviews (01-12-2012)“…The emerging class of RNA interference (RNAi) therapeutics is a fundamentally novel approach to treating human disease by enabling the pursuit of molecular…”
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randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus
Published in Proceedings of the National Academy of Sciences - PNAS (11-05-2010)“…RNA interference (RNAi) is a natural mechanism regulating protein expression that is mediated by small interfering RNAs (siRNA). Harnessing RNAi has potential…”
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Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate
Published in Molecular therapy (04-01-2017)“…Advancement of RNAi-based therapeutics depends on effective delivery to the site of protein synthesis. Although intravenously administered, multi-component…”
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ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients
Published in The Journal of heart and lung transplantation (01-02-2016)“…Background Respiratory syncytial virus (RSV) infection in lung transplant (LTx) patients is associated with an increased incidence of bronchiolitis obliterans…”
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IRAK4 degrader in hidradenitis suppurativa and atopic dermatitis: a phase 1 trial
Published in Nature medicine (01-12-2023)“…Toll-like receptor–driven and interleukin-1 (IL-1) receptor–driven inflammation mediated by IL-1 receptor–associated kinase 4 (IRAK4) is involved in the…”
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Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
Published in Orphanet journal of rare diseases (04-09-2015)“…Transthyretin-mediated amyloidosis is an inherited, progressively debilitating disease caused by mutations in the transthyretin gene. This study evaluated the…”
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Rapid progression of familial amyloidotic polyneuropathy: A multinational natural history study
Published in Neurology (25-08-2015)“…OBJECTIVES:To assess the association between severity of neuropathy and disease stage, and estimate the rate of neuropathy progression in a retrospective…”
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RNA Interference Therapy in Lung Transplant Patients Infected with Respiratory Syncytial Virus
Published in American journal of respiratory and critical care medicine (15-02-2011)“…Lower respiratory tract infections due to respiratory syncytial virus (RSV) are associated with development of bronchiolitis obliterans syndrome in lung…”
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Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy
Published in BMC neurology (11-09-2017)“…Patisiran is an investigational RNA interference (RNAi) therapeutic in development for the treatment of hereditary ATTR (hATTR) amyloidosis, a progressive…”
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First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement
Published in Cancer discovery (01-04-2013)“…RNA interference (RNAi) is a potent and specific mechanism for regulating gene expression. Harnessing RNAi to silence genes involved in disease holds promise…”
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A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
Published in Orphanet journal of rare diseases (08-07-2020)“…Abstract Background Patisiran, an RNA interference therapeutic, has demonstrated robust reduction of wild-type and mutant transthyretin protein and was able to…”
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Role of Raf Kinase in Cancer: Therapeutic Potential of Targeting the Raf/MEK/ERK Signal Transduction Pathway
Published in Seminars in oncology (01-08-2006)“…Improvements in our understanding of the molecular basis of cancer have led to the clinical development of protein kinase inhibitors, which target pivotal…”
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Phase II Trial of Sorafenib Plus Interferon Alfa-2b As First- or Second-Line Therapy in Patients With Metastatic Renal Cell Cancer
Published in Journal of clinical oncology (01-08-2007)“…We undertook this study to determine the activity and tolerability of sorafenib administered with interferon alfa-2b (IFN-alpha-2b) as first- or second-line…”
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Safety, Pharmacokinetics, and Activity of ABX-EGF, a Fully Human Anti–Epidermal Growth Factor Receptor Monoclonal Antibody in Patients With Metastatic Renal Cell Cancer
Published in Journal of clinical oncology (01-08-2004)“…To determine the antitumor activity of ABX-EGF, a fully human monoclonal antibody to the epidermal growth factor receptor (EGFr), in previously treated…”
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Phase I Trial of Sequential Low-Dose 5-Aza-2′-Deoxycytidine Plus High-Dose Intravenous Bolus Interleukin-2 in Patients with Melanoma or Renal Cell Carcinoma
Published in Clinical cancer research (01-08-2006)“…Purpose: The silencing of gene expression through DNA methylation contributes to defects in antigen presentation and apoptosis in melanoma and renal cell…”
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Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells
Published in PloS one (01-09-2016)“…Familial amyloid polyneuropathy (FAP) is caused by mutations of the transthyretin (TTR) gene, predominantly expressed in the liver. Two compounds that…”
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Decitabine Up-regulates S100A2 Expression and Synergizes with IFN-γ to Kill Uveal Melanoma Cells
Published in Clinical cancer research (01-09-2007)“…Purpose: Metastatic uveal melanoma is resistant to conventional chemotherapy and immunotherapy. In this study, we investigated the responsiveness of uveal…”
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603 Targeted STAT3 degradation leads to remodeling of an immunosuppressive tumor microenvironment and subsequent sensitization to immune checkpoint therapy
Published in Journal for immunotherapy of cancer (01-11-2021)“…BackgroundSignal Transducer and Activator of Transcription 3 (STAT3), a multifaceted transcription factor, is aberrantly activated across a variety of…”
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Phase I Trial of Concurrent Twice-Weekly Recombinant Human Interleukin-12 Plus Low-Dose IL-2 in Patients With Melanoma or Renal Cell Carcinoma
Published in Journal of clinical oncology (01-07-2003)“…To maintain interferon gamma (IFNgamma) induction by recombinant human interleukin-12 (rhIL-12) and enhance its activity against melanoma and renal cell…”
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