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Journal of Biological Chemistry, ISSN 0021-9258, 07/2010, Volume 285, Issue 29, pp. 22639 - 22650
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 8/2014, Volume 111, Issue 31, pp. 11449 - 11454
Nanoparticle-based experimental therapeutics are currently being investigated in numerous human clinical trials. CALAA-01 is a targeted, polymer-based... 
Nanoparticles | Animals | Humans | Medical treatment | Liver | Small interfering RNA | Pretreatment | Dosage | Polymers | Radiotherapy | Maximum tolerance dose | Dose limiting toxicity | DNA replication | DNA proliferation | Translational medicine | maximum tolerance dose | translational medicine | SYSTEMIC DELIVERY | DESIGN | RNAI | dose limiting toxicity | MULTIDISCIPLINARY SCIENCES | INHIBITORS | TRANSLATION | Liver - pathology | Demography | Kidney - pathology | Species Specificity | Middle Aged | Polyethylene Glycols - adverse effects | Male | Nanoparticles - adverse effects | Polyethylene Glycols - therapeutic use | Complement System Proteins - metabolism | Polymers - therapeutic use | Liver - drug effects | Nanoparticles - therapeutic use | Aged, 80 and over | Female | Polymers - adverse effects | Cytokines - blood | Immunization | Kidney - drug effects | RNA, Small Interfering - pharmacokinetics | Polyethylene Glycols - pharmacokinetics | RNA, Small Interfering - adverse effects | Rats | Treatment Outcome | Haplorhini | Platelet Count | Blood Coagulation - drug effects | RNA, Small Interfering - therapeutic use | Polymers - pharmacokinetics | Aged | Mice | Drugs | Pharmaceutical research | Drug targeting | Research | Genetic translation | Health aspects | Kidneys | Correlation analysis | Toxicity | Rodents | Ribonucleic acid--RNA | Index Medicus | Biological Sciences
Journal Article
Lancet, The, ISSN 0140-6736, 2014, Volume 383, Issue 9911, pp. 60 - 68
Summary Background Proprotein convertase subtilisin/kexin type 9 (PCSK9) binds to LDL receptors, leading to their degradation. Genetics studies have shown that... 
Internal Medicine | MEDICINE, GENERAL & INTERNAL | DENSITY-LIPOPROTEIN CHOLESTEROL | DOUBLE-STRANDED-RNA | PLASMA-CHOLESTEROL | METAANALYSIS | CARDIOVASCULAR-DISEASE | MONOCLONAL-ANTIBODY | HYPERCHOLESTEROLEMIA | STATINS | AMG 145 | ATORVASTATIN | Single-Blind Method | Serine Endopeptidases - biosynthesis | Humans | Middle Aged | RNA, Small Interfering - pharmacology | RNA, Small Interfering - adverse effects | Cholesterol, LDL - drug effects | Male | Healthy Volunteers | Proprotein Convertases - genetics | Dose-Response Relationship, Drug | Proprotein Convertases - biosynthesis | RNA Interference | Serine Endopeptidases - blood | Serine Endopeptidases - genetics | Adult | Cholesterol, LDL - blood | Female | Proprotein Convertases - blood | RNA, Small Interfering - administration & dosage | Genetic Therapy - adverse effects | Genetic Therapy - methods | Proprotein Convertase 9 | Enzymes | Research | Gene mutations | Properties | Gene expression | Identification and classification | Medical research | RNA | Low density lipoproteins | Anticholesteremic agents | Clinical trials | Aluminum compounds | Coronary heart disease | Cholesterol | Hypercholesterolemia | Analysis | Medicine, Experimental | Product development | Trans fatty acids | Blood proteins | Studies | Heart | Nanoparticles | Plasma | Nutrition research | Cardiovascular disease | Lipids | Mutation | Drug dosages | Statins | Index Medicus | Abridged Index Medicus
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 07/2018, Volume 379, Issue 1, pp. 11 - 21
Hereditary transthyretin amyloidosis is caused by the deposition of misfolded transthyretin proteins in peripheral nerves and other tissues. This phase 3 trial... 
MEDICINE, GENERAL & INTERNAL | POLYNEUROPATHY | EFFICACY | TAFAMIDIS | SAFETY | LATE-ONSET | NATURAL-HISTORY | PROGRESSION | Prealbumin - genetics | Humans | Middle Aged | Male | Amyloid Neuropathies, Familial - therapy | Infusions, Intravenous - adverse effects | Aged, 80 and over | Adult | Female | Prealbumin - analysis | Severity of Illness Index | Double-Blind Method | Amyloid Neuropathies, Familial - complications | RNA, Small Interfering - adverse effects | Polyneuropathies - therapy | Disease Progression | Administration, Intravenous | Polyneuropathies - etiology | RNA, Small Interfering - therapeutic use | Least-Squares Analysis | Quality of Life | Aged | RNAi Therapeutics | Amyloid Neuropathies, Familial - blood | Edema - chemically induced | Walk Test | Gait Disorders, Neurologic - etiology | Care and treatment | Amyloidosis | Genetic aspects | Intravenous administration | Gait | Cardiomyopathy | RNA-mediated interference | Liver | Diabetes mellitus | Body weight | Transthyretin | Diabetic neuropathy | Neuropathy | Gene expression | Polyneuropathy | Quality of life | Medicine | Neurology | Hospitals | Nutritional status | Pharmaceuticals | Index Medicus | Abridged Index Medicus | Assaigs clínics | Clinical trials | RNA | Amiloïdosi | Clinical Medicine | Neurologi | Medical and Health Sciences | Klinisk medicin | Medicin och hälsovetenskap
Journal Article
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 04/2017, Volume 376, Issue 15, pp. 1430 - 1440
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 01/2017, Volume 376, Issue 1, pp. 41 - 51
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2013, Volume 21, Issue 5, pp. 973 - 985
Journal Article
Journal Article