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Biology of Blood and Marrow Transplantation, ISSN 1083-8791, 2010, Volume 16, Issue 2, pp. 157 - 168
Journal Article
Bone Marrow Transplantation, ISSN 0268-3369, 07/2016, Volume 51, Issue 7, pp. 906 - 912
Journal Article
British Journal of Haematology, ISSN 0007-1048, 11/2013, Volume 163, Issue 4, pp. 444 - 457
Journal Article
Blood, ISSN 0006-4971, 03/2016, Volume 127, Issue 13, pp. 1656 - 1665
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2017, Volume 25, Issue 5, pp. 1069 - 1075
Oligonucleotides (oligos) have been under clinical development for approximately the past 30 years, beginning with antisense oligonucleotides (ASOs) and... 
Kynamro | therapeutics | Spinraza | Defitelio | Vitravene | Macugen | aptamer | phosphorothioate | Eteplirsen | oligonucleotide | MEDICINE, RESEARCH & EXPERIMENTAL | FAMILIAL HYPERCHOLESTEROLEMIA | VENOOCCLUSIVE DISEASE | B SYNTHESIS INHIBITOR | APOLIPOPROTEIN-B | LDL CHOLESTEROL | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | ENDOTHELIAL-CELLS | GENETICS & HEREDITY | DUCHENNE MUSCULAR-DYSTROPHY | EXTRACELLULAR-MATRIX | FIBROBLAST GROWTH-FACTORS | RECEPTOR-BINDING | Oligonucleotides - therapeutic use | Humans | Morpholinos - therapeutic use | Polydeoxyribonucleotides - therapeutic use | Drug Approval | Hepatic Veno-Occlusive Disease - therapy | Cytomegalovirus Retinitis - virology | Muscular Atrophy, Spinal - genetics | Hypercholesterolemia - pathology | Oligonucleotides, Antisense - therapeutic use | Hepatic Veno-Occlusive Disease - genetics | Hypercholesterolemia - therapy | Hepatic Veno-Occlusive Disease - pathology | Muscular Dystrophy, Duchenne - therapy | Cytomegalovirus Retinitis - therapy | Thionucleotides - therapeutic use | Aptamers, Nucleotide - therapeutic use | Muscular Dystrophy, Duchenne - pathology | Clinical Trials as Topic | Cytomegalovirus Retinitis - genetics | Muscular Atrophy, Spinal - pathology | Macular Degeneration - genetics | Macular Degeneration - therapy | Muscular Atrophy, Spinal - therapy | Hypercholesterolemia - genetics | Muscular Dystrophy, Duchenne - genetics | Macular Degeneration - pathology | Macular degeneration | Cytomegalovirus | Collaboration | Clinical trials | Antisense oligonucleotides | FDA approval | Mutation | Drug dosages | Binding sites | Marketing | Pharmaceuticals | Review
Journal Article
Lancet, The, ISSN 0140-6736, 2012, Volume 379, Issue 9823, pp. 1301 - 1309
Journal Article