Exosome and Nanoparticle Enabled Delivery of Genome Editors for Targeted Therapeutics

Engineered genome editors are transforming precision medicine, yet their clinical impact hinges on delivery: getting editing payloads to the right cells, at the right dose, at the right time—safely. Exosomes and synthetic nanoparticles have emerged as leading vehicles for nucleases, base/prime editors, and RNA-guided systems, offering tunable biodistribution, immune evasion, and modular cargo loading. This Research Topic invites interdisciplinary contributions advancing exosome- and nanoparticle-mediated delivery of genome editors toward effective, selective, and clinically translatable therapeutics.

Recent advances span endogenous exosome biogenesis engineering, ligand or aptamer-based tropism, and hybrid biomimetic vesicles that combine biological stealth with materials control. Parallel progress in lipid, polymer, and inorganic nanoparticles has improved endosomal escape, intracellular trafficking, and multiplexed cargo co-delivery (e.g., Cas protein, sgRNA, donor DNA, or mRNA). Despite momentum, key challenges remain: scalable and reproducible manufacturing, batch-to-batch consistency, navigating innate and adaptive immunity, minimizing off-target editing and biodistribution, and achieving durable on-target activity in difficult tissues.

We welcome all article types covering the following topics:

1. Vehicle engineering and targeting: exosome surface display, organ/tissue tropism, disease microenvironment targeting, hybrid vesicles, ligand- or antibody-guided nanoparticles.

2. Cargo strategies: RNPs vs mRNA/saRNA, base/prime editors, Cas variants and PAM-flexible tools, multi-cargo co-packaging, stimuli-responsive or logic-gated release.

3. Biological barriers and mechanisms: endosomal escape, nuclear transport, intracellular trafficking, immune sensing, RES clearance, mucus/ECM penetration.

4. Safety and specificity: off-target reduction, transient vs persistent expression, immunogenicity profiling, genotoxicity, integration risk, and mitigation frameworks.

5. Manufacturing and analytics: scalable bioproduction and purification of exosomes, GMP-ready nanoparticle synthesis, potency and identity assays, real-time QC, stability and shelf-life.

6. In vivo performance: pharmacokinetics/pharmacodynamics, biodistribution mapping, dose–response, repeat dosing, tissue and cell-type selectivity across preclinical models and early clinical studies.

7. Disease applications: oncology (tumor- and stroma-targeted editing), cardiovascular, neurodegenerative and CNS delivery, metabolic and muscle disorders, ocular and hepatic indications, and ex vivo/in situ strategies.

8. Computation and AI: design–build–test loops for vehicle and cargo optimization, predictive models for targeting/escape, multi-omics readouts of on/off-target outcomes.

Ethical, regulatory, and translational considerations are especially encouraged, including standardized assays, reporting guidelines, and risk–benefit frameworks for first-in-human studies, as well as data sharing and reproducibility practices to accelerate field-wide learning.

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Editorial
• FAIR² Data
• FAIR² DATA Direct Submission
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Editorial
• FAIR² Data
• FAIR² DATA Direct Submission
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Policy and Practice Reviews
• Policy Brief
• Review
• Systematic Review

Keywords: Exosome Therapeutics, CRISPR delivery, Extracellular Vesicles, Lipid Nanoparticles, Safety and Immunogenicity

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.