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Delivering mRNAs to mouse tissues using the SEND system
Abstract:
mRNAs produced in a cell are almost always translated within the same cell. Some mRNAs are transported to other cells of the organism through processes involving membrane nanotubes or extracellular vesicles. A recent report describes a surprising new phenomenon of encapsulating mRNAs inside virus-like particles (VLPs) to deliver them to other cells in a process that was named SEND (Selective Endogenous eNcapsidation for cellular Delivery). Although the seminal work demonstrates the SEND process in cultured cells, it is unknown whether this phenomenon occurs in vivo . Here, we demonstrate the SEND process in living organisms using specially designed genetically engineered mouse models. Our proof of principle study lays a foundation for the SEND-VLP system to potentially be used as a gene therapy tool to deliver therapeutically important mRNAs to tissues.
Insights
Researchers demonstrated a new mRNA delivery method, Selective Endogenous eNcapsidation for cellular Delivery (SEND), in living organisms. This virus-like particle (VLP) system shows potential for mRNA gene therapy applications in vivo.
Area of Science:
- Molecular Biology
- Gene Therapy
- Cell Biology
Background:
- Messenger RNAs (mRNAs) are typically translated within their cell of origin.
- Intercellular mRNA transport occurs via nanotubes or extracellular vesicles.
- A novel mRNA encapsulation and delivery mechanism, Selective Endogenous eNcapsidation for cellular Delivery (SEND), was recently reported in cultured cells.
Approach:
- Investigated the in vivo occurrence of the SEND process using genetically engineered mouse models.
- Developed and utilized specialized mouse models to validate endogenous mRNA encapsulation into virus-like particles (VLPs).
- Demonstrated the SEND phenomenon in a living organism context.
Key Points:
- The SEND process, previously observed in vitro, was successfully demonstrated in vivo.
- Virus-like particles (VLPs) can encapsulate and deliver endogenous mRNAs to other cells within a living organism.
- This study provides the first in vivo evidence of the SEND mechanism.
Conclusions:
- The SEND process occurs in vivo, extending beyond cell culture observations.
- The SEND-VLP system represents a potential platform for mRNA-based gene therapy.
- Further development of SEND-VLP technology could enable targeted mRNA delivery to specific tissues.
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