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Molecular Therapy, ISSN 1525-0016, 07/2017, Volume 25, Issue 7, pp. 1467 - 1475
Genetic drugs such as small interfering RNA (siRNA), mRNA, or plasmid DNA provide potential gene therapies to treat most diseases by silencing pathological... 
genetic drugs | gene therapy | lipid nanoparticles | siRNA | mRNA | gene editing | THERAPEUTICS | MEDICINE, RESEARCH & EXPERIMENTAL | MECHANISM | SIZE | ENCAPSULATION | SIRNA DELIVERY | CATIONIC LIPIDS | MESSENGER-RNA | POTENCY | FORMULATIONS | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | IN-VIVO | GENETICS & HEREDITY | RNA, Small Interfering - genetics | Hepatitis B - metabolism | Nanoparticles - chemistry | Hepatitis B - genetics | Amyloid Neuropathies, Familial - metabolism | Atherosclerosis - genetics | Humans | Hepatocytes - pathology | Antineoplastic Agents - therapeutic use | Liver Neoplasms - therapy | Hepatocytes - metabolism | Amyloid Neuropathies, Familial - therapy | RNA, Messenger - metabolism | Lipids - chemistry | Atherosclerosis - therapy | Carcinoma, Hepatocellular - genetics | RNA, Messenger - administration & dosage | Plasmids - genetics | Liver Neoplasms - pathology | Hepatocytes - drug effects | Atherosclerosis - pathology | Hepatitis B - therapy | Liver Neoplasms - genetics | Amyloid Neuropathies, Familial - genetics | RNA, Messenger - genetics | Hepatitis B - pathology | Clinical Trials as Topic | Antineoplastic Agents - chemistry | Plasmids - metabolism | Amyloid Neuropathies, Familial - pathology | Atherosclerosis - metabolism | Animals | Lipids - pharmacokinetics | Carcinoma, Hepatocellular - pathology | Liver Neoplasms - metabolism | Carcinoma, Hepatocellular - therapy | Nanoparticles - administration & dosage | RNA, Small Interfering - administration & dosage | Drug Delivery Systems - methods | Plasmids - administration & dosage | Carcinoma, Hepatocellular - metabolism | Genetic Therapy - methods | RNA, Small Interfering - metabolism | Drugs | Drug delivery systems | Ethanol | Advantages | Transthyretin | Lipids | Breast cancer | Ovarian cancer | Nanoparticles | Proteins | Gene silencing | Liver cancer | Hepatocytes | Plasmids | Amyloidosis | Polymers | Gene therapy | Deoxyribonucleic acid--DNA | Review
Journal Article
Orphanet Journal of Rare Diseases, ISSN 1750-1172, 12/2015, Volume 10, Issue 1, p. 109
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Journal of Neurology, Neurosurgery & Psychiatry, ISSN 0022-3050, 02/2012, Volume 83, Issue 2, pp. 152 - 158
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JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, ISSN 1085-9489, 03/2016, Volume 21, Issue 1, pp. 5 - 9
Transthyretin familial amyloid polyneuropathy (TTR-FAP) is a rare, progressive, life-threatening, hereditary disorder caused by mutations in the transthyretin... 
ATTR AMYLOIDOSIS | transthyretin | PITFALLS | diagnosis | NEUROSCIENCES | VAL30MET | CLINICAL NEUROLOGY | NEUROPATHY | FAP | hereditary amyloidosis | LATE-ONSET | MUTATIONS | transthyretin familial amyloid neuropathy | Early Diagnosis | Humans | Amyloid Neuropathies, Familial - diagnosis | Polyneuropathies | Amyloidosis | Genetic aspects | Carpal tunnel syndrome
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