Molecular therapy. Nucleic acidsMar 30, 2026

Tiny vesicles from acerola fruit for delivering gene-editing tools to the brain

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Jun 28, 2026

Abstract

Essence

Acerola-derived exosome-like nanovesicles enabled noninvasive CRISPR-Cas9 delivery to the central nervous system in a preclinical C9orf72 editing experiment.

Evidence

This delivery-platform study showed that peptide-tagged acerola exosome-like nanoparticle complexes carried Cas9-guide RNA ribonoproteins into GLP2-receptor-expressing neurons and achieved intranasal in vivo genome editing of C9orf72.

Caveat

The abstract describes a preclinical proof of delivery and editing rather than therapeutic outcomes, and its garbled disease-target details limit how precisely the result can be interpreted.

Simplified

An aberrant six-base repeat in intron 1 ofis the most frequent cause of solitary and familial amyotrophic lateral sclerosis and frontotemporal dementia. This mutation is a potential target for CRISPR/Cas9-based genome editing. However, the blood-brain barrier and limitations of current viral or nanoparticle-based delivery systems to neurons significantly restrict the clinical application of CRISPR-Cas9 in the brain. To address these challenges, we developed a drug delivery system using acerola-derived exosome-like nanoparticles (AELNs), which may overcome several limitations associated with human exosomes. AELNs stably form complexes with ribonucleoproteins (RNPs) comprised of Cas9 proteins and guide RNAs (gRNAs). We improved the delivery efficiency and selectivity of AELN/RNP complexes in GLP2-receptor-expressing neurons by incorporating GLP2 peptides into the AELN/RNP complexes. Intranasal administration of peptide-tagged AELN/RNP complexesconfirmed the successful genome editing of, demonstrating the potential of this system for treating neurodegenerative diseases. This study presents a potentially innovative approach forgenome editing using a noninvasive delivery system. C9orf72in vivo C9orf72in vivo