FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides
2017; Mary Ann Liebert, Inc.; Volume: 27; Issue: 2 Linguagem: Inglês
10.1089/nat.2017.0665
ISSN2159-3345
Autores Tópico(s)RNA modifications and cancer
ResumoNucleic Acid TherapeuticsVol. 27, No. 2 Issues in DevelopmentFDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating OligonucleotidesAnnemieke Aartsma-RusAnnemieke Aartsma-RusPublished Online:1 Apr 2017https://doi.org/10.1089/nat.2017.0665AboutSectionsView articleView Full TextPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail View articleFiguresReferencesRelatedDetailsCited byIntrathecal Pseudodelivery of Drugs in the Therapy of Neurodegenerative Diseases: Rationale, Basis and Potential Applications25 February 2023 | Pharmaceutics, Vol. 15, No. 3PROGRESS OF OLIGONUCLEOTIDE THERAPEUTICS TARGET TO RNA16 December 2022Mesyl Phosphoramidate Oligonucleotides: A New Promising Type of Antisense Agents19 January 2023Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy22 December 2022 | JCI Insight, Vol. 7, No. 24Nephrotoxicity of marketed antisense oligonucleotide drugsCurrent Opinion in Toxicology, Vol. 32Nano drug delivery systems for antisense oligonucleotides (ASO) therapeuticsJournal of Controlled Release, Vol. 352Antisense oligonucleotide induced pseudoexon skipping and restoration of functional protein for Fukuyama muscular dystrophy caused by a deep-intronic variant25 November 2022 | Human Molecular GeneticsGold Nanoparticle-Mediated Gene Therapy31 October 2022 | Cancers, Vol. 14, No. 21Clinical and Research Readiness for Spinal Muscular Atrophy: The Time Is Now for Knowledge Translation29 July 2022 | Physical Therapy, Vol. 102, No. 104′‐ C ‐Aminoethoxy‐Modified DNAs Exhibit Increased Nuclease Resistance, Sustained RNase H Activity, and Inhibition of KRAS Gene Expression30 June 2022 | Chemistry & Biodiversity, Vol. 19, No. 8Splicing mutations in the CFTR gene as therapeutic targets2 June 2022 | Gene Therapy, Vol. 29, No. 7-8Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients7 June 2022 | European Journal of Neurology, Vol. 29, No. 8RNA-based therapeutics: an overview and prospectus23 July 2022 | Cell Death & Disease, Vol. 13, No. 72′-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity Dmytro Honcharenko, Cristina S.J. 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