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Related Concept Videos

MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
MicroRNAs01:22

MicroRNAs

MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...

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Engineering Lipid Nanoparticles for mRNA Immunotherapy.

Robby Zwolsman1, Youssef B Darwish1, Ewelina Kluza1

  • 1Laboratory of Chemical Biology, Department of Biomedical Engineering, Eindhoven University of Technology, Eindhoven, the Netherlands.

Wiley Interdisciplinary Reviews. Nanomedicine and Nanobiotechnology
|April 8, 2025
PubMed
Summary
This summary is machine-generated.

Messenger RNA (mRNA) therapies, delivered via lipid nanoparticles (LNPs), show promise for various diseases. Further development is needed to optimize LNP-mRNA delivery for widespread therapeutic use.

Keywords:
immunotherapylipid nanoparticles (LNP)messenger RNA (mRNA)

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Area of Science:

  • Biotechnology
  • Immunology
  • Nanomedicine

Background:

  • Messenger RNA (mRNA) therapeutics offer a versatile platform for genetic instructions.
  • Chemical modifications and nanotechnology are crucial for mRNA stability and delivery.
  • Lipid nanoparticles (LNPs) are the leading delivery system for mRNA therapeutics.

Purpose of the Study:

  • To review the current state of LNP-mRNA technology for therapeutic applications.
  • To discuss the potential of LNP-mRNA in immunotherapy, including vaccination, oncology, and autoimmune disorders.
  • To identify challenges and future directions for LNP-mRNA development.

Main Methods:

  • Review of existing literature on LNP-mRNA technology.
  • Analysis of LNP-mRNA applications in vaccination and immunotherapy.
  • Discussion of challenges in LNP-mRNA delivery and immunostimulatory effects.

Main Results:

  • LNPs are highly effective for mRNA delivery, particularly for vaccines and hepatic applications.
  • LNP-mRNA holds significant potential for diverse immunotherapeutic strategies.
  • Key challenges include controlling biodistribution and mitigating immunostimulatory effects.

Conclusions:

  • LNP-mRNA technology is a rapidly advancing field with broad therapeutic potential.
  • Overcoming challenges in delivery and immunogenicity is critical for clinical translation.
  • Advanced methods like library screening and machine learning will drive future innovations.