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Cited by 222RNA Interference and Gene DeliveryNanoparticle-Based Drug DeliveryAdvancements in Transdermal Drug Delivery

Ionizable lipid nanoparticles for in utero mRNA delivery

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Riley, Rachel S., Kashyap, Meghana V., Billingsley, Margaret M., White, Brandon, Alameh, Mohamad-Gabriel, Bose, Sourav K., Zoltick, Philip W., Li, Hiaying +5 more·2021·Science Advances
DOI 10.1126/sciadv.aba1028PMC PMC7806221
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Abstract

Lipid nanoparticles deliver mRNA to mouse fetuses, which may ultimately enable in utero therapy to treat fetal genetic diseases.

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  • The 12-LNP library had hydrodynamic diameters ranging from 67.8 to 128.3 nm with a maximum polydispersity value of 0.154, indicating small and relatively monodisperse LNPs.

    “Hydrodynamic diameter for all LNP formulations – measured via dynamic light scattering intensity – ranged from 67.8 to 128.3 nm with a maximum polydispersity value of 0.154 (Fig. 1B), indicating small and relatively monodisperse LNPs (PDI < 0.3) (19).”

    From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [19]

    The Results section, “Characterization of the LNP library” (Fig. 2C; page not provided), reports 14 formulations with DLS-measured diameters of 64.6–135.2 nm, not 12 formulations ranging from 67.8–128.3 nm. It also says one formulation had PDI above 0.3, contradicting the claimed maximum of 0.154, although the others were described as monodisperse. (LLM, conf=0.94)

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  • The top-performing LNP following in utero ICV mRNA delivery was in the bottom quartile of LNPs for targeting the fetal liver following in utero intravascular mRNA delivery.

    “Importantly, the top-performing LNP following in utero ICV mRNA delivery was in the bottom quartile of LNPs for targeting the fetal liver following in utero intravascular mRNA delivery (19).”

    From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [19]

    The Results sections on in utero mRNA delivery and intravascular injection (page not available) compare LNP formulations after vitelline-vein injection and report fetal-liver delivery. Our copy does not report an ICV delivery experiment or identify an ICV top performer as falling in the bottom quartile for liver delivery, so the specific cross-route comparison is unsupported. (LLM, conf=0.84)

    Experimental — accuracy not yet measured

  • LNPs for mRNA delivery to the fetal mouse liver following in utero intravascular injection were previously identified.

    “We previously identified LNPs for mRNA delivery to the fetal mouse liver following in utero intravascular injection (19).”

    From Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease (2023) · cites this paper as [19]

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Authors
Riley, R. S., Kashyap, M. V., Billingsley, M. M., White, B., Alameh, M.-G., Bose, S. K., Zoltick, P. W., Li, H., Zhang, R., Cheng, A. Y., Weissman, D., Peranteau, W. H., & Mitchell, M. J.
Affiliations
  • University of Pennsylvania (US)
  • Children's Hospital of Philadelphia (US)
  • Abramson Cancer Center (US)
Corresponding author
  • Riley, Rachel S.
  • Kashyap, Meghana V.
  • Billingsley, Margaret M.
  • Peranteau, William H.
  • Mitchell, Michael J.
ORCID
Venue
Science Advances
Publisher
American Association for the Advancement of Science (AAAS)
Published
2021-01-15
Volume
7
Issue
3
ISSN
2375-2548 (ISSN-L)
DOI
10.1126/sciadv.aba1028
PMC
PMC7806221
OpenAlex
W3120748003
Cited by
222
References
10
Citations by year
2026: 23 · 2025: 49 · 2024: 50 · 2023: 49 · 2022: 31 · 2021: 19 · 2020: 1
FWCI
11.68
Type
journal article
Language
en
Open access
Licence
Locations
  • Science Advances (publishedVersion, cc-by-nc)
  • Europe PMC (PubMed Central) (publishedVersion, cc-by-nc)
  • PubMed Central (publishedVersion, cc-by-nc)
  • PubMed (publishedVersion)
  • Rowan Digitals Works (Rowan University) (submittedVersion, cc-by-nc-sa)
Funders
  • National Institutes of Health (DP2 TR002776, DP2HL152427, T32HL007954, F32HL143861, 1F32CA243475-01A1, 1R01DK123049-01)
  • Institute for Translational Medicine and Therapeutics (ITMAT) Transdisciplinary Program in Translational Medicine and Therapeutics
  • Burroughs Wellcome Fund (Career Award at the Scientific Interface)
  • Institute for Translational Medicine and Therapeutics (ITMAT) Transdisciplinary Program in Translational Medicine and Therapeutics.
  • Tau Beta Pi
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
  • Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
  • Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advancements in Transdermal Drug Delivery
Keywords
ionizable lipid nanoparticles, prenatal gene therapy, fetal liver, luciferase reporter, secreted proteins, genome editing, mouse fetus
MeSH terms
Gene Editing, Animals, Liposomes, RNA, Messenger, Mice, Nanoparticles

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