Extracellular Vesicle-Based Delivery System for Cancer and Neurodegenerative Disease Treatment

A special issue of Pharmaceutics (ISSN 1999-4923). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: 31 July 2025 | Viewed by 2178

Special Issue Editors


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Dipartimento di Medicina e Chirurgia, Università degli Studi di Milano-Bicocca, Piazza dell'Ateneo Nuovo, 1-20126 Milan, Italy
Interests: extracellular vesicles; cargo; cancer

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Guest Editor
Department of Oncology and Hematology-Oncology, Università degli Studi di Milano, 20122 Milano, Italy
Interests: nanovaccines; nanomaterials; cancer immunotherapy; nanomedicine; mRNA-based immunotherapy; drug and gene delivery; non-viral vectors
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Special Issue Information

Dear Colleagues,

We are pleased to invite you to contribute to our Special Issue on “Extracellular Vesicle-Based Delivery System for Cancer and Neurodegenerative Disease Treatment”.

Extracellular vesicles (EVs) have emerged as promising nanocarriers capable of crossing biological barriers and delivering therapeutic agents with high specificity and reduced toxicity. The most clinically approved delivery vehicles are represented by liposomes, which are EV-like structures sharing structural similarities with EVs but differing in composition and ease of production. EVs offer greater biological complexity and potential for drug loading but face manufacturing and quality control challenges. Future applications involve the use of EV-based platforms for the delivery of immunotherapeutics, such as mRNA-based drugs. Overall, the field of EVs could be critical for advancing targeted therapies, which can significantly contribute to improving the outcomes of patients with cancer or neurodegenerative conditions.

This Special Issue aims to gather innovative research and comprehensive reviews on the development and application of EV-based delivery systems. We seek to explore the mechanisms, challenges, and clinical potential of these systems (by EVs and EV-like structures), aligning with cutting-edge therapeutic strategies and drug/gene delivery technologies.

In this Special Issue, we welcome original research articles and reviews. Research areas may include (but are not limited to) the engineering of EVs for drug delivery, therapeutic applications in cancer and neurodegenerative diseases, and clinical translation of EV-based therapies.

We look forward to receiving your contributions.

Dr. Eva Costanzi
Dr. Stefano Persano
Prof. Dr. Stefano Leporatti
Guest Editors

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Keywords

  • extracellular vesicles
  • liposomes
  • cancer
  • neurodegenerative diseases
  • immunotherapy
  • mRNA delivery
  • drug delivery system
  • therapeutics

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Published Papers (2 papers)

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Review

20 pages, 1129 KiB  
Review
Harnessing Extracellular Vesicles for Targeted Drug Delivery in Ovarian Cancer
by Jang-Hyuk Yun, Yoo Rim Noh, Seongkyeong Yoo, Soohyun Park, Yunsup Choi, Jiyeon An and Iljin Kim
Pharmaceutics 2025, 17(4), 528; https://doi.org/10.3390/pharmaceutics17040528 - 17 Apr 2025
Viewed by 358
Abstract
Ovarian cancer remains one of the most lethal gynecologic malignancies, primarily due to late-stage diagnosis, high recurrence rates, and the development of chemoresistance. Although targeted therapies have improved patient outcomes, their efficacy is often limited by off-target toxicity and acquired drug resistance. Extracellular [...] Read more.
Ovarian cancer remains one of the most lethal gynecologic malignancies, primarily due to late-stage diagnosis, high recurrence rates, and the development of chemoresistance. Although targeted therapies have improved patient outcomes, their efficacy is often limited by off-target toxicity and acquired drug resistance. Extracellular vesicles (EVs), nanoscale vesicles naturally released by cells, have emerged as promising carriers for precision drug delivery. This review provides a comprehensive overview of recent advances in EV-based therapeutic strategies for ovarian cancer, including the delivery of chemotherapeutic agents, nucleic acid therapeutics, and immunomodulatory molecules. We further explore innovative engineering approaches to enhance targeting specificity, such as surface modification, cell source selection, biomaterial integration, and magnetic nanoparticle-assisted delivery. Key translational challenges in bringing EV-based therapies to clinical application are also addressed. Collectively, these insights underscore the transformative potential of EV-based platforms in advancing targeted and personalized treatment for ovarian cancer. Full article
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29 pages, 2805 KiB  
Review
Molecular Targeting of Ischemic Stroke: The Promise of Naïve and Engineered Extracellular Vesicles
by Jihun Lee, Dongho Geum, Dong-Hyuk Park and Jong-Hoon Kim
Pharmaceutics 2024, 16(12), 1492; https://doi.org/10.3390/pharmaceutics16121492 - 21 Nov 2024
Cited by 1 | Viewed by 1202
Abstract
Ischemic stroke (IS) remains a leading cause of mortality and long-term disability worldwide, with limited therapeutic options available. Despite the success of early interventions, such as tissue-type plasminogen activator administration and mechanical thrombectomy, many patients continue to experience persistent neurological deficits. The pathophysiology [...] Read more.
Ischemic stroke (IS) remains a leading cause of mortality and long-term disability worldwide, with limited therapeutic options available. Despite the success of early interventions, such as tissue-type plasminogen activator administration and mechanical thrombectomy, many patients continue to experience persistent neurological deficits. The pathophysiology of IS is multifaceted, encompassing excitotoxicity, oxidative and nitrosative stress, inflammation, and blood–brain barrier disruption, all of which contribute to neural cell death, further complicating the treatment of IS. Recently, extracellular vesicles (EVs) secreted naturally by various cell types have emerged as promising therapeutic agents because of their ability to facilitate selective cell-to-cell communication, neuroprotection, and tissue regeneration. Furthermore, engineered EVs, designed to enhance targeted delivery and therapeutic cargo, hold the potential to improve their therapeutic benefits by mitigating neuronal damage and promoting neurogenesis and angiogenesis. This review summarizes the characteristics of EVs, the molecular mechanisms underlying IS pathophysiology, and the emerging role of EVs in IS treatment at the molecular level. This review also explores the recent advancements in EV engineering, including the incorporation of specific proteins, RNAs, or pharmacological agents into EVs to enhance their therapeutic efficacy. Full article
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