Title: | The Pharmacokinetics of 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides: Experiences from Developing Exon Skipping Therapies for Duchenne Muscular Dystrophy |
Journal : | Nucleic acid therapeutics |
Authors: | Bosgra S, Author ; Sipkens J ; De Kimpe S ; den Besten C ; Datson N ; van Deutekom J |
Material Type: | Article |
Publisher: | United States, 08/2019 |
Languages: | English |
Keywords : | 2'-O-methyl phosphorothioate oligonucleotide ; antisense oligonucleotides ; Duchenne muscular dystrophy ; gene therapy ; neuromuscular disease ; pharmacokinetics ; review article |
Keywords: | 2'-O-methyl phosphorothioate;Duchenne muscular dystrophy;drisapersen;oligonucleotides;pharmacokinetics; |
Pubmed / DOI : | Pubmed : 31429628 / DOI : 10.1089/nat.2019.0805 |
N° Profil MNM : | 2019091 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31429628 |