Review
Pharmacology & Pharmacy
Hojun Choi, Kyungsun Choi, Dae-Hwan Kim, Byung-Koo Oh, Hwayoung Yim, Soojin Jo, Chulhee Choi
Summary: Delivering therapeutics to the central nervous system (CNS) is difficult due to the blood-brain barrier (BBB). Different mechanisms, such as receptor-mediated transcytosis, can be utilized for therapeutic delivery across the BBB. Exosomes, as natural nanoparticles, have the potential to cross the BBB and can be surface-engineered. Surface-modified exosomes have shown enhanced CNS delivery in preclinical studies, but the development of clinically approved exosome therapeutics for CNS diseases requires addressing the challenges of monitoring exosomal delivery to the brain parenchyma in vivo and understanding the mechanisms of BBB crossing.
Article
Chemistry, Physical
Soodeh Salarpour, Mahmood Barani, Abbas Pardakhty, Mehrdad Khatami, Narendra Pal Singh Chauhan
Summary: Brain-derived exosomes are nanovesicles released by central nervous system cells and play a crucial role in communication and waste management in the brain. They have been identified as important factors in Alzheimer's disease, Parkinson's disease, multiple sclerosis, and glioblastoma, and their ability to cross the blood-brain barrier offers potential for drug delivery and non-invasive monitoring of brain diseases.
JOURNAL OF MOLECULAR LIQUIDS
(2022)
Review
Cell & Tissue Engineering
Linli Li, Peipei Wu, Hui Qian, Wenrong Xu, Hui Shi, Jiajia Jiang
Summary: Extracellular vesicles (EVs) have small size, low immunogenicity and toxicity, and can carry a variety of bioactive molecules, making them essential in tissue injury and regeneration. However, challenges such as insufficient therapeutic efficacy, lack of targeting ability, and low yield have been observed in the application of EVs. Engineering and modification of EVs, as well as the introduction of targeting peptides, antibodies, and nanoparticles, can enhance their therapeutic efficacy and targeting ability. Additionally, the use of extracellular vesicle mimetics (EMs) and EV-mimicking nanocomplexes can improve production and simplify purification. Combining EVs with biomaterials further enhances their therapeutic efficacy and regenerative outcome.
STEM CELLS INTERNATIONAL
(2022)
Article
Chemistry, Multidisciplinary
Jun-Yong Wu, Yong-Jiang Li, Xiong-Bin Hu, Si Huang, Shilin Luo, Tiantian Tang, Da-Xiong Xiang
Summary: The study compared Exos and BNVs for brain tumor drug delivery, finding that BNVs derived from brain-derived endothelial cells are a promising alternative with higher yield and similar drug-loading capacity to natural exosomes. Despite different mechanisms, BNV/DOX showed similar therapeutic effects to Exo/DOX in inhibiting the progression of glioblastoma, with minimal systemic toxicity. The findings suggest that autologous BNVs are an effective alternative to Exos for brain tumor nanomedicine.
JOURNAL OF CONTROLLED RELEASE
(2021)
Review
Pharmacology & Pharmacy
Bhakti Pawar, Nupur Vasdev, Tanisha Gupta, Mahi Mhatre, Anand More, Neelima Anup, Rakesh Kumar Tekade
Summary: This article discusses the limitations of the blood-brain barrier (BBB) on brain drug delivery and ways to overcome it using new techniques such as nanomedicine, nose-to-brain drug delivery, and peptides as drug delivery carriers. It also helps to understand the factors affecting BBB permeability and the body clearance of drugs delivered to the brain.
Article
Biotechnology & Applied Microbiology
Jun-Yong Wu, Yong-Jiang Li, Jiemin Wang, Xiong-Bin Hu, Si Huang, Shilin Luo, Da-Xiong Xiang
Summary: The multifunctional DTX@Ang-EM significantly inhibit the growth of orthotopic GBM with reduced side effects of chemotherapy, providing a new strategy for targeted brain drug delivery and GBM therapy.
JOURNAL OF NANOBIOTECHNOLOGY
(2021)
Article
Pharmacology & Pharmacy
Negin Mozafari, Hajar Ashrafi, Amir Azadi
Summary: Neuroinflammation is a key feature of neurodegenerative disorders, and research on drug delivery systems and treatments targeting the immune system is crucial for controlling brain diseases associated with neuroinflammation.
JOURNAL OF DRUG DELIVERY SCIENCE AND TECHNOLOGY
(2021)
Review
Biochemistry & Molecular Biology
Panagiotis Skouras, Antonios N. Gargalionis, Christina Piperi
Summary: Exosomes are small extracellular vesicles that play a crucial role in cellular communication by transmitting signals between cells. They are involved in various physiological and pathological processes, including tumor progression and neurodegenerative diseases. Gliomas, in particular, secrete exosomes that influence cell invasion, migration, immune tolerance, and resistance to treatment. These exosomes carry molecules that promote communication between cancer cells and surrounding cells, providing valuable information about the tumor. Engineered exosomes also show potential as a drug delivery system. This review discusses the role of exosomes in glioma pathogenesis, their diagnostic utility, and potential applications in treatment.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Chemistry, Multidisciplinary
Tengfei Zhu, Ming Kong, Yingying Yu, Manfred Schartl, Deborah Mary Power, Chen Li, Wenxiu Ma, Yanxu Sun, Shuo Li, Bowen Yue, Weijing Li, Changwei Shao
Summary: Exosomes are cell-derived vesicles that have advantages in biocompatibility and engineerability. This study expanded the applications of engineered exosomes to sex-determining gene studies in early vertebrates. By developing an exosome-based delivery system, the researchers efficiently regulated the dmrt1 gene and discovered its important roles in the testis of juvenile fish.
JOURNAL OF CONTROLLED RELEASE
(2023)
Article
Chemistry, Multidisciplinary
Hongshuai Zhang, Xin Zheng, Tao Zhao, Yiping Chen, Yang Luo, Yangcan Dong, Hao Tang, Jianhui Jiang
Summary: Exosomes secreted from cells can serve as a potential biomarker for disease investigation. A dual-nanopore biosensor using DNA aptamers to recognize CD63 protein on exosomes is developed for label-free detection. The biosensor allows sensitive detection with a limit of 3.4 x 10(6) particles/mL. By trapping a single cell in a confined microwell, the biosensor successfully monitors exosome secretion from a single cell in different cell lines and under different stimuli.
Article
Respiratory System
Tess Kramer, Lizzy Wijmans, Martijn de Bruin, Ton van Leeuwen, Teodora Radonic, Peter Bonta, Jouke T. Annema
Summary: Bronchoscopic nCLE imaging of suspected peripheral lung cancer is feasible and safe, allowing real-time detection of lung cancer and accurate differentiation between malignancy and airway/lung parenchyma. Blinded raters showed high accuracy in distinguishing nCLE videos of lung tumors, indicating the potential of nCLE imaging as a real-time guidance tool.
Review
Biochemistry & Molecular Biology
Yuan Jiang, Fengbo Wang, Ke Wang, Yongqiang Zhong, Xiaofei Wei, Qiongfen Wang, Hong Zhang
Summary: Exosomes are natural membrane vesicles released from cells that play an important role in cell-cell communication. They have been found to have various roles in brain diseases and have received attention as therapeutic drug carriers for brain drug delivery. Engineered exosomes have shown potential in improving drug uptake efficiency and efficacy. However, challenges still exist in developing exosomes as a drug delivery strategy for brain diseases. Further research in this area is important for its development.
CURRENT MEDICINAL CHEMISTRY
(2022)
Article
Chemistry, Multidisciplinary
Bei Liu, Lirong Sun, Xijian Lu, Yuping Yang, Hongshang Peng, Zhaogang Sun, Juan Xu, Hongqian Chu
Summary: This study presents a metal-organic framework (MOF)-based nanotheranostic system that enables real-time drug release monitoring and combined chemo-photothermal therapy. By encapsulating photothermal agents (CuS) and therapeutic drug (DOX), the system achieves effective treatment while allowing drug release behaviors to be monitored in real-time. Moreover, the addition of folic acid-conjugated polyethylene glycol (FA-PEG) antennas enhances the active targeting effect of the system towards cancer cells.
Article
Engineering, Industrial
Tianxiang Li, Tielin Shi, Zirong Tang, Guanglan Liao, Jian Duan, Jinghui Han, Zhaopeng He
Summary: This paper presents a smart cutting tool embedded with thin-film thermocouple for real-time temperature monitoring in harsh cutting environments. The tool showed good sensitivity, dynamic response, and functional validity, and was able to reflect the progression of tool wear based on cutting temperature. The devised smart tool is useful for determining optimum cutting parameters and extending tool life.
JOURNAL OF MATERIALS PROCESSING TECHNOLOGY
(2021)
Article
Behavioral Sciences
Raghuram Kandimalla, Mohd Saeed, Neetu Tyagi, Ramesh C. Gupta, Farrukh Aqil
Summary: Alzheimer's disease (AD) is a widely studied neurological disorder that affects millions of individuals worldwide and is caused by misfolded proteins in the brain, particularly amyloid-beta and tau. While therapeutic interventions and nanotechnological advancements can help manage AD symptoms, a cure for this disease has yet to be found. Exosomes, which can originate from various cell and tissue types, have a dual role in the progression and treatment of AD. They can be manipulated to deliver therapeutic cargos, both small molecules and macromolecules, to the brain. This review focuses on the roles of exosomes in the pathology, diagnosis, and treatment of AD, as well as their potential as drug carriers for AD treatment.
NEUROSCIENCE AND BIOBEHAVIORAL REVIEWS
(2023)
Article
Biochemistry & Molecular Biology
Elena Barbon, Charlotte Kawecki, Solenne Marmier, Aboud Sakkal, Fanny Collaud, Severine Charles, Giuseppe Ronzitti, Caterina Casari, Olivier D. Christophe, Cecile V. Denis, Peter J. Lenting, Federico Mingozzi
Summary: Von Willebrand disease (VWD), the most common inherited bleeding disorder, has limited efficacy with current liver-directed gene therapy. This study developed a gene therapy strategy using an optimized AAV vector and endothelial-targeting peptide to deliver the VWF gene into endothelial cells, achieving long-term expression.
Editorial Material
Biotechnology & Applied Microbiology
Sem J. Aronson, Giuseppe Ronzitti, Piter J. Bosma
EXPERT OPINION ON BIOLOGICAL THERAPY
(2023)
Review
Clinical Neurology
E. Berling, R. Nicolle, P. Laforet, G. Ronzitti
Summary: Gene therapy, a therapeutic approach involving genetic material as a drug, is used to treat diseases, including neurological disorders. Different gene therapy approaches have varying mechanisms of action and levels of maturity. This review focuses on gene therapy techniques applied to Duchenne muscular dystrophy (DMD) and discusses gene transfer techniques, DNA editing, and RNA-level approaches. Clinical data and the advantages, limitations, and risks associated with gene therapy are presented.
REVUE NEUROLOGIQUE
(2023)
Article
Multidisciplinary Sciences
Ramachandran Prakasam, Angela Bonadiman, Roberta Andreotti, Emanuela Zuccaro, Davide Dalfovo, Caterina Marchioretti, Debasmita Tripathy, Gianluca Petris, Eric N. Anderson, Alice Migazzi, Laura Tosatto, Anna Cereseto, Elena Battaglioli, Gianni Soraru, Wooi Fang Lim, Carlo Rinaldi, Fabio Sambataro, Naemeh Pourshafie, Christopher Grunseich, Alessandro Romanel, Udai Bhan Pandey, Andrea Contestabile, Giuseppe Ronzitti, Manuela Basso, Maria Pennuto
Summary: Prakasam and colleagues demonstrate that targeting overexpressed co-activators Lsd1 and Prmt6 with artificial miRNAs can attenuate polyQ-expanded androgen receptor toxicity, ameliorating spinal-bulbar muscular atrophy phenotypes in flies and mice. This study suggests that targeting these co-regulators could be a potential therapeutic strategy for patients with SBMA, as it helps mitigate toxic gain-of-function without exacerbating loss-of-function mechanisms.
NATURE COMMUNICATIONS
(2023)
Review
Chemistry, Medicinal
Nicola Salvatore Orefice, Rossella Di Raimo, Davide Mizzoni, Mariantonia Logozzi, Stefano Fais
Summary: This article introduces how to combine biotechnology and organic agriculture to overcome the main challenges in drug delivery systems. The role of organic agriculture in future human health treatment is still a binary organic-conventional question, but exosomes-like nanoparticles provide a new organic path that plants and vegetables can release. The article proposes plant-derived exosome-like nanovesicles and discusses their important biological and pharmacological roles, representing a new tool for drug delivery.
EXPERT OPINION ON THERAPEUTIC PATENTS
(2023)
Review
Oncology
Mariantonia Logozzi, Nicola Salvatore Orefice, Rossella Di Raimo, Davide Mizzoni, Stefano Fais
Summary: Exosomes, nanosized extracellular vesicles, have been studied for their role in tumor pathogenesis and progression, as well as a new source of tumor biomarkers. Clinical studies have shown that exosome levels in plasma are higher in tumor patients and that plasma exosomes express well-known tumor markers and nucleic acids. The technical methods for obtaining and characterizing exosomes include Nanosight Tracking Analysis, immunocapture-based ELISA, and nano-scale flow cytometry. The acidic tumor microenvironment influences both the quantity and characteristics of exosomes released by tumor cells.
Article
Medicine, Research & Experimental
Michela Lisjak, Alessandra Iaconcig, Corrado Guarnaccia, Antonio Vicidomini, Laura Moretti, Fanny Collaud, Giuseppe Ronzitti, Lorena Zentilin, Andres F. Muro
Summary: Gene therapy has shown promising results in treating citrullinemia type I in mice, with the potential to prolong survival and improve symptoms, especially in neonates.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2023)
Meeting Abstract
Biotechnology & Applied Microbiology
Helena Costa Verdera, Monah Abou Alezz, Vasco Meneghini, Elisabeth Mangiameli, Inderpal Singh, Klaudia Kuranda, Ivan Merelli, Giuseppe Ronzitti, Angela Gritti, Federico Mingozzi, Anna Kajaste-Rudnitski
Meeting Abstract
Biotechnology & Applied Microbiology
Duohao Ren, Audrey Pineiro, Gaelle Chaveau, Julie Vendomele, Divya Ail, Deniz Dalkara, Giuseppe Ronzitti, Anne Galy, Sylvain Fisson
Meeting Abstract
Biotechnology & Applied Microbiology
Miquel Garcia, Sergio Munoz, Joan Bertolin, Veronica Jimenez, Maria Luisa Jaen, Anna Pujol, Laia Vila, Victor Sacristan, Elena Barbon, Giuseppe Ronzitti, Jihad El Andari, Warut Tulalamba, Quang Hong Pham, Jesus Ruberte, Thierry C. Vandendriessche, Marinee K. L. Chuah, Dirk Grimm, Federico Mingozzi, Fatima Bosch
Meeting Abstract
Biotechnology & Applied Microbiology
Antoine Gardin, Jeremy Rouillon, Valle Montalvo-Romeral, Lucille Rossiaud, Patrice Vidal, Romain Launay, Mallaury Vie, Berangere Bertin, Guillaume Dubreuil, Justine Nozi, Louisa Jauze, Romain Fragnoud, Nathalie Daniele, Laetitia Van Wittenberghe, Jeremy Esque, Isabelle Andre, Xavier Nissan, Lucile Hoch, Giuseppe Ronzitti
Meeting Abstract
Biotechnology & Applied Microbiology
Louisa Jauze, Mallaury Vie, Quentin Miagoux, Margot Jarrige, Patrice Vidal, Maria del Valle Montalvo-Romeral, Lucille Rossiaud, Justine Nozi, Pierre-Romain Le Brun, Jeremy Rouillon, Fanny Collaud, Emilie Bertil-Froidevaux, Christophe Georger, Laetitia van Wittenberghe, Adeline Miranda, Nathalie Daniele, Lucile Hoch, Xavier Nissan, Giuseppe Ronzitti
Meeting Abstract
Biotechnology & Applied Microbiology
Tiziana La Bella, Berangere Bertin, Ante Mihaljevic, Dylan Serillon, Anastasia Barkova, Justine Nozi, Patrice Vidal, Jean-Philippe Buffet, Sandrine Imbeaud, Jean-Charles Nault, Julien Cottineau, Jessica Zucman-Rossi, Giuseppe Ronzitti
Meeting Abstract
Biotechnology & Applied Microbiology
Marine Laurent, Christine Jenny, Julie Oustelandt, Anais Brassier, Francesca Landini, Consiglia Pacelli, Giuseppe Ronzitti, Mario Amendola
Correction
Clinical Neurology
B. Souchet, M. Audrain, Y. Gu, M. F. Lindberg, N. S. Orefice, E. Rey, N. Cartier, N. Janel, L. Meijer, Jerome Braudeau
JPAD-JOURNAL OF PREVENTION OF ALZHEIMERS DISEASE
(2023)