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Molecular Therapy, ISSN 1525-0016, 10/2019, Volume 27, Issue 10, pp. 1749 - 1757
Cystic fibrosis (CF) is an autosomal recessive monogenic disease caused by mutations in the gene. Therapeutic approaches that are focused on correcting CFTR... 
Journal Article
Journal of the American College of Cardiology, ISSN 0735-1097, 03/2018, Volume 71, Issue 11, pp. A658 - A658
Journal Article
Proceedings of the National Academy of Sciences of the United States of America, ISSN 0027-8424, 10/2018, Volume 115, Issue 43, p. 11066
Centronuclear myopathies (CNM) are a group of severe muscle diseases for which no effective therapy is currently available. We have previously shown that... 
Phenotypes | Size distribution | Genetic disorders | Oligonucleotides | Muscles | Viruses | Antisense oligonucleotides | Injection | Skeletal muscle | Proteins | Genotype & phenotype | Reduction | Histopathology | Ultrastructure | Dynamin | Mice | Mutation | Guanosinetriphosphatase | Myopathy
Journal Article
Journal of Cystic Fibrosis, ISSN 1569-1993, 05/2019, Volume 18, Issue 3, p. 334
The epithelial sodium channel ENaC consists of three subunits encoded by Scnn1a, Scnn1b, and Scnn1g and increased sodium absorption through this channel is... 
Medical research | Messenger RNA | Analysis | Mucins | Medicine, Experimental | Cystic fibrosis
Journal Article
Neurodegenerative disease management, 02/2019, Volume 9, Issue 1, pp. 25 - 30
Journal Article
Nucleic Acid Therapeutics, ISSN 2159-3337, 04/2019, Volume 29, Issue 2, pp. 82 - 91
Kallikrein is the key contact system mediator responsible for the conversion of high-molecular-weight kininogen into the inflammatory vasodilator peptide... 
hereditary angioedema | antisense oligonucleotide | plasma prekallikrein
Journal Article
Blood, ISSN 0006-4971, 12/2014, Volume 124, Issue 21, pp. 2812 - 2812
Abstract Cell-associated and circulating hemostatic factors have been identified as powerful determinants of tumor cell metastatis, primarily by supporting the... 
Journal Article
Journal Article
Molecular Cancer Therapeutics, ISSN 1535-7163, 12/2015, Volume 14, Issue 12 Supplement 2, pp. A73 - A73
Journal Article
Blood, ISSN 0006-4971, 12/2014, Volume 124, Issue 21, pp. LBA-1 - LBA-1
Abstract BACKGROUND: Patients undergoing total knee arthroplasty are at risk for postoperative venous thromboembolism (VTE). The pathogenesis of postoperative... 
Journal Article
Journal of Cystic Fibrosis, ISSN 1569-1993, 05/2019, Volume 18, Issue 3, pp. 334 - 341
The epithelial sodium channel ENaC consists of three subunits encoded by , , and and increased sodium absorption through this channel is hypothesized to lead... 
Cystic fibrosis | ENaC | Subunit | Antisense oligonucleotide | CONDUCTANCE | AMILORIDE | EPITHELIAL SODIUM-CHANNEL | TRIAL | NA+ CHANNEL | RESPIRATORY SYSTEM | LUNG-DISEASE | RESPIRATORY EPITHELIA | POTENTIAL DIFFERENCE | EXPRESSION | CFTR
Journal Article
RSC Drug Discovery Series, ISSN 2041-3203, 2019, Volume 2019-, Issue 68, pp. 62 - 79
Journal Article
Molecular Vision, ISSN 1090-0535, 01/2017, Volume 23, p. 561
Purpose: Age-related macular degeneration (AMD) is the leading cause of permanent vision loss among the elderly in many industrialized countries, and the... 
Macular degeneration | Proteins | Plasma | Complement activation | Liver | Complement factor B | Antisense oligonucleotides | mRNA | Alternative pathway | Age | Geriatrics
Journal Article
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