A5011250594- Hepatitis C virus research
- RNA Interference and Gene Delivery
- Blood Coagulation and Thrombosis Mechanisms
- Hemophilia Treatment and Research
- Hemoglobinopathies and Related Disorders
- MicroRNA in disease regulation
- Iron Metabolism and Disorders
- Cancer-related gene regulation
- HIV/AIDS drug development and treatment
- Pneumocystis jirovecii pneumonia detection and treatment
- Advanced biosensing and bioanalysis techniques
- Hepatitis B Virus Studies
- Trace Elements in Health
- Cytokine Signaling Pathways and Interactions
- Porphyrin Metabolism and Disorders
- Epigenetics and DNA Methylation
- Genetic and phenotypic traits in livestock
- Peptidase Inhibition and Analysis
- Influenza Virus Research Studies
- Lung Cancer Research Studies
- Wildlife Ecology and Conservation
- CRISPR and Genetic Engineering
- Kidney Stones and Urolithiasis Treatments
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Viral Infections and Immunology Research
Alnylam Pharmaceuticals (United States)
2011-2019
AbbVie (Germany)
2016-2018
Framingham State University
2016
In recent years, RNA interference (RNAi) therapeutics, most notably with lipid nanoparticle-based delivery systems, have advanced into human clinical trials. The results from these early trials suggest that nanoparticles (LNPs), and the novel ionizable lipids comprise them, will be important materials in this emerging field of medicine. A persistent theme use for biomedical applications has been incorporation biodegradability as a means to improve biocompatibility and/or facilitate...
Significance The safe, selective, and efficient delivery of siRNA is a key challenge to the broad application therapeutics in humans. Motivated by structure lipoproteins, we developed lipopeptide nanomaterials for delivery. In vivo mice, siRNA–lipopeptide particles provide most potent hepatocytes (ED 50 ∼ 0.002 mg/kg FVII silencing), with highest selectivity over nontarget cell types (orders magnitude), yet reported. These materials also show efficacy nonhuman primates.
Primary hyperoxaluria type 1 (PH1), an inherited rare disease of glyoxylate metabolism, arises from mutations in the enzyme alanine-glyoxylate aminotransferase. The resulting deficiency this leads to abnormally high oxalate production calcium crystal formation and deposition kidney many other tissues, with systemic oxalosis ESRD being a common outcome. Although small subset patients manages vitamin B6 treatments, only effective treatment for most is combined liver-kidney transplant, which...
Therapeutics based on RNA interference (RNAi) have emerged as a potential new class of drugs for treating human disease by silencing the target messenger (mRNA), thereby reducing levels corresponding pathogenic protein. The major challenge RNAi therapeutics is development safe delivery vehicles small interfering RNAs (siRNAs). We previously showed that cholesterol-conjugated siRNAs (chol-siRNA) associate with plasma lipoprotein particles and distribute primarily to liver after systemic...
Summary Real‐world studies are relevant to complement clinical trials on novel antiviral therapies against chronic hepatitis C; however, practice data currently limited. This study investigated effectiveness and safety of ombitasvir/paritaprevir/ritonavir ( OBV / PTV /r)±dasabuvir DSV )±ribavirin RBV ) for treatment HCV genotype GT 1 4 infection in a large real‐world cohort. The German Hepatitis C Registry is an observational cohort prospectively collecting direct‐acting therapies. Patients...
Diminished β-globin synthesis in β-thalassemia is associated with ineffective erythropoiesis, leading to secondary iron overload caused by inappropriately low levels of hepcidin and splenomegaly the symptomatic thalassemias. Splenectomy often employed patients reduce hemolysis. Expression regulatory peptide hormone repressed serine protease TMPRSS6. Hepcidin induction RNAi-mediated inhibition TMPRSS6 expression reduces mitigates anemia murine models intermedia. To interrogate efficacy...
Revusiran is a 1st-generation short interfering RNA targeting transthyretin conjugated to an N-acetylgalactosamine ligand facilitate delivery hepatocytes via uptake by the asialoglycoprotein receptors. Revusiran, in development for treatment of hereditary transthyretin-mediated amyloidosis, was discontinued after imbalance deaths "ENDEAVOUR" phase 3 clinical trial. Nonclinical safety assessments included pharmacology, acute and repeat-dose toxicity, genotoxicity, carcinogenicity. There were...
Populations of Hippopotamus amphibius have declined throughout Africa in recent years, and are expected to decline further. An understanding the population genetics individual populations hippos is necessary for effective management. To that end, we sequenced a portion mitochondrial DNA (mtDNA) control region or D-loop from 37 H. amphibius, six herds central Kruger National Park (KNP), Republic South Africa. We amplified 453 bp segment by PCR, identified 21 polymorphic sites seven...
Einleitung: Ca. 300.000 Patienten in Deutschland leiden an chronischer Hepatitis C. Das direkt antivirale Regime aus Ombitasvir (OBV), Paritaprevir (entwickelt von AbbVie und Enanta, co-dosiert mit Ritonavir [PTV/r]) Dasabuvir (DSV) mit/ohne Ribavirin (RBV) wurde 2015 zugelassen zur Behandlung C vom Genotyp 1 (GT1) erreichte Raten eines anhaltend virologischen Ansprechens (SVR12) zwischen 95% 100% klinischen Zulassungsstudien.
Background: Chronic hepatitis C virus (HCV) infection is highly prevalent in patients with a history of intravenous drug abuse; however historically, implementation HCV treatment this patient group real-world remained challenging. With all-oral, interferon-free regimens available and the favorable setting opiate substitution (OST), conditions are now potentially more adequate for successful these patients. In clinical trials OST chronic genotype 1 (GT1) infection, regimen ombitasvir (OBV),...
Background: Chronic hepatitis C virus (HCV) infection is common in patients with renal insufficiency and end-stage disease, but clinical data on HCV therapy this patient group limited. The direct-acting antiviral regimen of ombitasvir (OBV), paritaprevir (co-dosed ritonavir, PTV/r) dasabuvir (DSV) or without ribavirin (RBV) was approved 2015 Germany for treatment chronic genotype 1 (GT1) 4 (GT4) infection. However, real-world disease currently