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Related Experiment Video

Updated: Apr 29, 2026

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

Lipid nanoparticles for bone marrow-targeted RNA therapeutics.

L D Mata Casimiro¹, R Escudero¹, M du Plessis¹

¹Prinses Maxima Centrum for Pediatric Oncology, Heidelberglaan 25, 3584 CS, Utrecht, The Netherlands.

Journal of Nanobiotechnology

|April 27, 2026

Summary

Lipid nanoparticles (LNPs) show promise for treating bone marrow (BM) diseases, but face delivery challenges. Innovative LNP designs and targeting strategies are needed to improve BM accumulation for effective RNA therapies.

Keywords:

Bone marrow Gene therapy Lipid nanoparticle (LNP)RNA therapeutics Targeting

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Published on: June 18, 2018

Area of Science:

Biomedical Engineering
Nanotechnology
Hematology

Background:

Lipid nanoparticles (LNPs) are crucial for RNA delivery, particularly for bone marrow (BM) applications like leukemia treatment and regenerative therapies.
Current LNP formulations show limited accumulation in the BM after systemic administration due to physiological barriers.
These barriers include rapid clearance, liver sequestration, complement activation, and poor endosomal escape, hindering extrahepatic therapeutic efficacy.

Purpose of the Study:

To review the challenges and strategies for enhancing LNP delivery to the bone marrow.
To characterize BM physiology and its barriers to LNP accumulation.
To highlight innovative LNP designs and targeting approaches for improved BM-specific RNA therapeutics.

Main Methods:

Literature review of LNP delivery to the bone marrow.
Analysis of BM physiology and barriers to nanoparticle accumulation.
Evaluation of passive and active targeting strategies for LNPs.
Assessment of preclinical in vitro models and LNP design innovations.

Main Results:

Bone marrow delivery of therapeutic RNA via LNPs faces significant obstacles.
Physiological barriers like mononuclear phagocyte system clearance and liver sequestration limit LNP BM accumulation.
Innovative strategies in LNP design, stability, circulation time, and targeting are emerging.

Conclusions:

Overcoming BM delivery barriers is essential for realizing the potential of LNP-based RNA therapeutics.
Next-generation LNPs require enhanced stability, circulation, and targeted delivery for BM-associated diseases.
Advances in LNP design and understanding of BM physiology pave the way for novel treatments.