AN ARTICLE FROM IMMUNOSTEP

Exosomes and CRISPR: Targeted gene editing in cancer and fibrosis.

Immunostep recently published an article titled “Exosomes and CRISPR: Targeted gene editing in cancer and fibrosis.” Exosomes, small extracellular vesicles of 30–150 nm, have moved beyond being simple mediators of intercellular communication. One of the most innovative applications in recent laboratory research is their use as delivery vectors for exosomes and CRISPR/Cas9 systems, offering a non-viral and highly selective approach for investigating gene editing in preclinical models of cancer and liver fibrosis.

What are Exosomes as CRISPR/Cas9 Delivery Platforms?

The CRISPR/Cas9 system has revolutionized biomedicine due to its ability to perform precise DNA cuts, but its application in the lab is often limited by challenges in efficient delivery and cell specificity. Viral vectors, commonly used in research, carry risks of immunogenicity, mutagenesis, and complex manufacturing. Here, exosomes emerge as a disruptive alternative for in vitro and in vivo studies:

• They can be efficiently loaded with guide RNA (sgRNA) and Cas9 mRNA/protein.

• They exhibit low immunogenicity in standard immunocompetent models, as they are derived from host cells.

• Their natural tropism can be engineered to target specific cell types, such as tumor cells or hepatic stellate cells during research assays.

What are the Preclinical Applications in Cancer?

In oncology research, CRISPR-functionalized exosomes are studied to target key genes involved in tumor proliferation and survival. They are also used to modulate the tumor microenvironment in preclinical models, enabling highly specific targeted research.