The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

Akin Akinc, Martin A. Maier, Muthiah Manoharan, Kevin Fitzgerald, Muthusamy Jayaraman, Scott Baros, Steven Ansell, Xinyao Du, Michel J. Hope, Thomas D. Madden, Barbara L. Mui, Sean C. Semple, Ying K. Tam, Marco A. Ciufolini, Dominik Witzigmann, Jayesh A. Kulkarni, Roy van der Meel, Pieter R. Cullis (Corresponding author)

Onderzoeksoutput: Bijdrage aan tijdschriftCommentaar/Brief aan de redacteurAcademicpeer review

16 Citaten (Scopus)

Samenvatting

The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many nucleic acid-based therapies enabled by nanoparticle delivery.
Originele taal-2Engels
Pagina's (van-tot)1084-1087
Aantal pagina's4
TijdschriftNature Nanotechnology
Volume14
Nummer van het tijdschrift12
DOI's
StatusGepubliceerd - 4 dec 2019

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Citeer dit

Akinc, A., Maier, M. A., Manoharan, M., Fitzgerald, K., Jayaraman, M., Baros, S., ... Cullis, P. R. (2019). The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs. Nature Nanotechnology, 14(12), 1084-1087. https://doi.org/10.1038/s41565-019-0591-y