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A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

Akinc, Akin, Zumbuehl, Andreas, Goldberg, Michael, Leshchiner, Elizaveta S, Busini, Valentina, Hossain, Naushad, Bacallado, Sergio A, Nguyen, David N, Fuller, Jason, Alvarez, Rene, Borodovsky, Anna, Borland, Todd, Constien, Rainer, de Fougerolles, Antonin, Dorkin, J Robert, Narayanannair Jayaprakash, K, Jayaraman, Muthusamy, John, Matthias, Koteliansky, Victor, Manoharan, Muthiah, Nechev, Lubomir, Qin, June, Racie, Timothy, Raitcheva, Denitza, Rajeev, Kallanthottathil G, Sah, Dinah W Y, Soutschek, Jürgen, Toudjarska, Ivanka, Vornlocher, Hans-Peter, Zimmermann, Tracy S, Langer, Robert, Anderson, Daniel G·2008·Nature Biotechnology
DOI 10.1038/nbt1402
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Abstract

The safe and effective delivery of RNA interference (RNAi) therapeutics remains an important challenge for clinical development. The diversity of current delivery materials remains limited, in part because of their slow, multi-step syntheses. Here we describe a new class of lipid-like delivery molecules, termed lipidoids, as delivery agents for RNAi therapeutics. Chemical methods were developed to allow the rapid synthesis of a large library of over 1,200 structurally diverse lipidoids. From this library, we identified lipidoids that facilitate high levels of specific silencing of endogenous gene transcripts when formulated with either double-stranded small interfering RNA (siRNA) or single-stranded antisense 2'-O-methyl (2'-OMe) oligoribonucleotides targeting microRNA (miRNA). The safety and efficacy of lipidoids were evaluated in three animal models: mice, rats and nonhuman primates. The studies reported here suggest that these materials may have broad utility for both local and systemic delivery of RNA therapeutics.

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Crossref · 10.1038/nbt1402

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4 of 4 references verified0 citation errors · 0 not checkable

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  • Delivery of therapeutic RNA molecules (siRNA or microRNA) encapsulated within lipid or lipid-like carriers into cells or tissues is effective in infectious diseases and tumors.

    “The delivery of therapeutic RNA molecules (siRNA or microRNA) encapsulated within the lipid or lipid-like carrier231,232 into the cells or tissues was proven to be effective in infectious diseases and tumors, as shown in many preclinical studies233,234,235 and clinical trials (reviewed by Kaczmarek et al.236).”

    From MiR-128-3p – a gray eminence of the human central nervous system (2024) · cites this paper as [231]

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Research works printed but not linked: 30

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Metadata

Authors
Akinc, A., Zumbuehl, A., Goldberg, M., Leshchiner, E. S., Busini, V., Hossain, N., Bacallado, S. A., Nguyen, D. N., Fuller, J., Alvarez, R., Borodovsky, A., Borland, T., Constien, R., de Fougerolles, A., Dorkin, J. R., Narayanannair Jayaprakash, K., Jayaraman, M., John, M., Koteliansky, V., Manoharan, M., Nechev, L., Qin, J., Racie, T., Raitcheva, D., Rajeev, K. G., Sah, D. W. Y., Soutschek, J., Toudjarska, I., Vornlocher, H.-P., Zimmermann, T. S., Langer, R., & Anderson, D. G.
Venue
Nature Biotechnology
Publisher
Springer Science and Business Media LLC
Published
2008-04-27
Volume
26
Issue
5
Pages
561-569
ISSN
  • 1087-0156
  • 1546-1696
DOI
10.1038/nbt1402
Cited by
1,150
References
4
Language
en
Licence

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