Cargando…
Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
Nucleic acid‐based therapeutics that regulate gene expression have been developed towards clinical use at a steady pace for several decades, but in recent years the field has been accelerating. To date, there are 11 marketed products based on antisense oligonucleotides, aptamers and small interferin...
Autores principales: | Hammond, Suzan M, Aartsma‐Rus, Annemieke, Alves, Sandra, Borgos, Sven E, Buijsen, Ronald A M, Collin, Rob W J, Covello, Giuseppina, Denti, Michela A, Desviat, Lourdes R, Echevarría, Lucía, Foged, Camilla, Gaina, Gisela, Garanto, Alejandro, Goyenvalle, Aurelie T, Guzowska, Magdalena, Holodnuka, Irina, Jones, David R, Krause, Sabine, Lehto, Taavi, Montolio, Marisol, Van Roon‐Mom, Willeke, Arechavala‐Gomeza, Virginia |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2021
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8033518/ https://www.ncbi.nlm.nih.gov/pubmed/33821570 http://dx.doi.org/10.15252/emmm.202013243 |
Ejemplares similares
-
Delivery is key: lessons learnt from developing splice‐switching antisense therapies
por: Godfrey, Caroline, et al.
Publicado: (2017) -
Splicing modulation therapy in the treatment of genetic diseases
por: Arechavala-Gomeza, Virginia, et al.
Publicado: (2014) -
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides
por: Aartsma-Rus, Annemieke, et al.
Publicado: (2023) -
Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides
por: Goyenvalle, Aurélie, et al.
Publicado: (2023) -
Antisense-Mediated RNA Targeting: Versatile and Expedient Genetic Manipulation in the Brain
por: Zalachoras, Ioannis, et al.
Publicado: (2011)