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Related Experiment Video

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Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy
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PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery.

Bingxin Liu1, Yamato Sajiki2, Nicole Littlefield3

  • 1Perinatal Institute, Division of Neonatology, Perinatal, and Pulmonary Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA; Materials Science and Engineering Program, Department of Mechanical and Materials Engineering, University of Cincinnati College of Medicine, Cincinnati, OH 45221, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|January 27, 2025
PubMed
Summary
This summary is machine-generated.

New lipid nanoparticles (LNPs) effectively deliver messenger RNA (mRNA) to specific lung cells in vivo. Poly(β-amino ester)s (PBAEs) show promise for targeted gene therapy in lung diseases.

Keywords:
PBAE-PEGcell-specificlipid nanoparticlelung endothelial cellslung epithelial cells

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Area of Science:

  • Biotechnology
  • Nanomedicine
  • Gene Therapy

Background:

  • Messenger RNA (mRNA) delivered via lipid nanoparticles (LNPs) is a successful vaccination strategy.
  • Targeted delivery of mRNA to specific lung cell types using LNPs remains a challenge.
  • Poly(β-amino ester)s (PBAEs) are a class of polymers with potential for LNP formulation.

Purpose of the Study:

  • To evaluate the efficacy of PBAE and PEGylated PBAE (PBAE-PEG) based LNPs for mRNA delivery into distinct lung cell types in vivo.
  • To compare the transfection efficiency of PBAE-based LNPs with commercial transfection reagents.
  • To assess the impact of mRNA modification (5-Methoxyuridine) on transfection efficiency.

Main Methods:

  • Formulation of LNPs using PBAE or PBAE-PEG with specific lipid components (4A3-SC8/DOPE/cholesterol/DOTAP).
  • In vitro transfection efficiency assessment in HEK293T cells using PBAE-PEG/LNP and Lipofectamine MessengerMAX.
  • In vivo mRNA delivery studies in mice via intravenous (IV) and intratracheal (IT) administration, followed by cell-type specific transfection analysis.

Main Results:

  • PBAE-PEG/LNP demonstrated comparable in vitro transfection efficiency to Lipofectamine MessengerMAX.
  • In vivo, intravenous PBAE-PEG/LNP achieved 73% mRNA transfection in lung endothelial cells.
  • Intratracheal PBAE-PEG/LNP achieved 55% transfection in lung alveolar type II (ATII) cells, while PBAE/LNP showed superior delivery to airway club cells.
  • 5-Methoxyuridine-modified mRNA enhanced in vivo transfection but not in vitro.

Conclusions:

  • PBAE-PEG/LNP and PBAE/LNP formulations are effective for delivering mRNA to multiple lung cell types in vivo.
  • These PBAE-based LNPs offer a viable platform for gene therapy applications targeting genetic lung diseases.
  • The choice of LNP composition and administration route can be optimized for specific lung cell targeting.