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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 Enhance mRNA Delivery to the Central Nervous System Upon Intrathecal Injection.

Yonger Xue^1,2, Chang Wang^1,2, Haoyuan Li¹

¹Icahn Genomics Institute, Precision Immunology Institute, Department of Immunology and Immunotherapy, Icahn School of Medicine at Mount Sinai, New York, NY, 10029, USA.

Advanced Materials (Deerfield Beach, Fla.)

|May 3, 2025

Summary

New brain-targeting lipid nanoparticles (BLNPs) effectively deliver messenger RNA (mRNA) to the central nervous system (CNS) via intrathecal injection, showing promise for treating neurological disorders.

Keywords:

central nervous system intrathecal injection lipid nanoparticles mRNA

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Area of Science:

Biotechnology
Neuroscience
Drug Delivery

Background:

Lipid nanoparticle-messenger RNA (LNP-mRNA) formulations show therapeutic promise but face challenges in central nervous system (CNS) delivery.
Effective mRNA delivery to the brain is crucial for treating neurological diseases.

Purpose of the Study:

To develop and evaluate brain-targeting lipid nanoparticles (BLNPs) for efficient mRNA delivery to the CNS.
To assess the efficacy and safety of BLNPs in preclinical models.

Main Methods:

Development of BLNPs by incorporating brain-targeting small molecules with amino lipids and helper lipids.
Intrathecal injection of BLNP-mRNA formulations (e.g., GFP, Cre recombinase, Cas9 mRNA/sgRNA) in mouse models.
Evaluation of mRNA delivery, gene expression in neurons and astrocytes, and genome editing efficiency.
Comparison of BLNP safety and biocompatibility with FDA-approved lipid nanoparticles (LNPs).

Main Results:

The lead TD5 BLNP formulation demonstrated superior mRNA delivery to the brain compared to DLin-MC3-DMA LNPs upon intrathecal injection.
TD5 BLNPs facilitated significant GFP and tdTomato expression in both neurons and astrocytes across major brain regions.
Effective genome editing in the brain was achieved using Cas9 mRNA/sgRNA delivered by TD5 BLNPs.
TD5 BLNPs exhibited comparable safety and biocompatibility profiles to MC3 LNPs.

Conclusions:

TD5 BLNPs represent a promising strategy for effective mRNA delivery to brain tissues via intrathecal administration.
This formulation enables efficient gene expression in both neuronal and glial cells, offering potential for CNS disease therapeutics.
The developed BLNPs overcome a significant hurdle in CNS-targeted mRNA delivery, paving the way for novel neurological treatments.