Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

• Published in: EMBO molecular medicine Vol. 13; no. 4; pp. e13243 - n/a
• Main Authors: 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
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 09.04.2021; John Wiley & Sons, Inc; EMBO Press; Wiley Open Access; John Wiley and Sons Inc; Springer Nature
• Subjects: Antisense oligonucleotides; Antisense therapy; Aptamers; Chemistry; delivery; Deoxyribonucleic acid; DNA; Drug development; EMBO08; EMBO36; EMBO41; Gene expression; Genes; Homeopathy; Life Sciences; Lipids; Materia medica and therapeutics; Metabolism; MicroRNAs; Molecular weight; Nanoparticles; Nucleic acids; oligonucleotides; Peptides; Polyethylene glycol; preclinical models; Protein expression; Proteins; Regulatory approval; Review; RNA therapeutics; safety; Therapeutics; United States > US; delivery; preclinical models; oligonucleotides; RNA therapeutics; safety

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...