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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs.
Akinc A, Maier MA, Manoharan M, Fitzgerald K, Jayaraman M, Barros S, Ansell S, Du X, Hope MJ, Madden TD, Mui BL, Semple SC, Tam YK, Ciufolini M, Witzigmann D, Kulkarni JA, van der Meel R, Cullis PR. Akinc A, et al. Among authors: witzigmann d. Nat Nanotechnol. 2019 Dec;14(12):1084-1087. doi: 10.1038/s41565-019-0591-y. Nat Nanotechnol. 2019. PMID: 31802031 No abstract available.
The Biomolecular Corona of Lipid Nanoparticles for Gene Therapy.
Francia V, Schiffelers RM, Cullis PR, Witzigmann D. Francia V, et al. Among authors: witzigmann d. Bioconjug Chem. 2020 Sep 16;31(9):2046-2059. doi: 10.1021/acs.bioconjchem.0c00366. Epub 2020 Aug 31. Bioconjug Chem. 2020. PMID: 32786370 Review.
In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA.
Villiger L, Rothgangl T, Witzigmann D, Oka R, Lin PJC, Qi W, Janjuha S, Berk C, Ringnalda F, Beattie MB, Stoffel M, Thöny B, Hall J, Rehrauer H, van Boxtel R, Tam YK, Schwank G. Villiger L, et al. Among authors: witzigmann d. Nat Biomed Eng. 2021 Feb;5(2):179-189. doi: 10.1038/s41551-020-00671-z. Epub 2021 Jan 25. Nat Biomed Eng. 2021. PMID: 33495639 Free PMC article.
56 results