Article
Medicine, Research & Experimental
Junquera Lopez-Seijas, Diego Miranda-Balbuena, Alba Iglesias-Fente, Marta Sacristan-Santos, Natalia Carballo-Pedrares, Maria C. Arufe, Ana Rey-Rico, Juan Fafian-Labora
Summary: Senescence is an irreversible cell-cycle arrest process that is closely related to aging and age-related diseases. Gene therapy has emerged as a powerful tool for treating such diseases, but genetic modification of senescent cells is challenging. Niosomes, self-assembled nanocarriers, show great potential as an alternative for genetic modification of senescent cells due to their cytocompatibility and versatility. In this study, niosomes were used for the first time to genetically modify senescent umbilical cord-derived mesenchymal stem cells, and the results showed that niosome composition greatly influenced transfection efficiency, with formulations containing sucrose and cholesterol being the most suitable. Furthermore, the resulting niosome formulations exhibited higher transfection efficiency and lower cytotoxicity compared to the commercial reagent Lipofectamine. These findings highlight the potential of niosomes as effective vectors for genetic modification of senescent cells, offering new possibilities for the prevention and treatment of age-related diseases.
MOLECULAR THERAPY-NUCLEIC ACIDS
(2023)
Review
Pharmacology & Pharmacy
Hui Zu, Danchen Gao
Summary: Although non-viral vectors in gene therapy have low cytotoxicity and immunogenicity, they still face challenges in gene transfer efficiency, specificity, gene expression duration, and safety. Overcoming these obstacles is necessary to enhance the effectiveness of non-viral vectors in gene therapy.
Review
Biochemistry & Molecular Biology
Federica Staurenghi, Michelle E. McClements, Ahmed Salman, Robert E. MacLaren
Summary: Non-viral gene therapy has the potential to overcome limitations of viral vector-based therapeutics. In recent years, methods of in vivo plasmid delivery have been developed, but further improvements are needed. Minicircles, as DNA vectors, have the advantage of limiting immune responses, and can be a viable option for treating inherited retinal diseases. Optimizing their delivery to the neural retina remains a challenge.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Pharmacology & Pharmacy
Nuseibah AL Qtaish, Idoia Gallego, Ilia Villate- Beitia, Myriam Sainz-Ramos, Gema Martinez-Navarrete, Cristina Soto-Sanchez, Eduardo Fernandez, Patricia Galvez-Martin, Tania B. Lopez-Mendez, Gustavo Puras, Jose Luis Pedraz
Summary: Compared to niosomes, niosphingosomes and their corresponding complexes showed decreased particle size and increased superficial charge. Cell viability and transfection efficiency increased when cells were exposed to niosphingoplexes, although there were no significant differences in cellular uptake. Niosphingoplexes exhibited decreased endocytosis via caveolae, higher co-localization with the lysosomal compartment, and enhanced endosomal escape properties.
EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS
(2021)
Article
Pharmacology & Pharmacy
Hardy Mitdank, Simko Sama, Meike Troeger, Maria Francesca Testa, Mattia Ferrarese, Dario Balestra, Mirko Pinotti, Alexander Weng
Summary: Minicircle DNA is a promising tool in gene therapy with advantages such as higher transfection efficiency and longer expression time, but its cost-intensive production limits broader use. An optimized production method can lead to high-quality minicircle DNA, which has been investigated for pharmaceutical potential in vitro in a tumor cell model.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2021)
Article
Chemistry, Multidisciplinary
Yi Yan, Xiao-Yu Liu, An Lu, Xiang-Yu Wang, Lin-Xia Jiang, Jian-Cheng Wang
Summary: RNA-based therapy is a promising strategy for disease treatment, and recent advances in non-viral delivery systems have shown potential in protecting RNA, facilitating cell internalization, and controlled release of therapeutics.
JOURNAL OF CONTROLLED RELEASE
(2022)
Review
Biotechnology & Applied Microbiology
Kai Ma, Chun-Liu Mi, Xiang-Xiang Cao, Tian-Yun Wang
Summary: Gene delivery systems are crucial for gene therapy and recombinant protein production, with non-viral vector gene delivery reagents offering advantages such as large packaging load capacity and low immunogenicity. Liposomes and non-liposome cationic polymers are commonly used as gene delivery reagents, with different head structures including quaternary ammonium salt, amine, amino acid, polypeptide, guanidine salt, and a heterocyclic ring.
APPLIED MICROBIOLOGY AND BIOTECHNOLOGY
(2021)
Review
Biotechnology & Applied Microbiology
Laurens Raes, Stefaan C. De Smedt, Koen Raemdonck, Kevin Braeckmans
Summary: Genetically engineered T cells have shown promising potential in cancer treatment, with a focus on CAR T cell therapies. The next generation of CAR T cell therapies will require more complex engineering strategies and may involve revolutionary gene editing approaches. Non-viral transfection technologies are gaining interest as alternatives to viral vectors, with the potential to enhance the manufacturing process and improve safety considerations.
BIOTECHNOLOGY ADVANCES
(2021)
Review
Pharmacology & Pharmacy
Antoine Hakim, Benjamin Guido, Lokesh Narsineni, Ding-Wen Chen, Marianna Foldvari
Summary: Glaucoma is a progressive disease caused by the death of retinal ganglion cells and axonal loss in the optic nerve. Elevated intraocular pressure is a major risk factor contributing to this condition. Current management mainly focuses on lowering intraocular pressure, but it does not address optic nerve degeneration. Gene therapy, especially non-viral delivery systems, shows promise in controlling genes involved in glaucoma pathophysiology and providing neuroprotection.
ADVANCED DRUG DELIVERY REVIEWS
(2023)
Review
Polymer Science
Elena Cojocaru, Jana Ghitman, Raluca Stan
Summary: Gene-based therapy is a cutting-edge approach in medical biotechnology that aims to introduce genetic material into specific cells and tissues to stimulate or inhibit signaling pathways. The integration of gene delivery technologies with electrospun fibrous architectures offers a promising strategy for improving stability and local delivery of therapeutic genes.
Review
Pharmacology & Pharmacy
Ahmed Salman, Ariel Kantor, Michelle E. McClements, Gemma Marfany, Sonia Trigueros, Robert E. MacLaren
Summary: The development of the CRISPR/Cas system has revolutionized molecular biology and offers great potential for gene therapy. The retina, as a target for gene therapy, has advantages such as surgical accessibility and relative immune privilege. However, viral vector-mediated therapies have limitations, and the use of nanomaterials provides an alternative approach.
Article
Materials Science, Multidisciplinary
Jing Chen, Wen Nie, Yuzhu Hu, Yangmei Shen, Yunzhu Lin, Bilan Wang, Zhiyong Qian, Xiang Gao
Summary: Drug resistance is a challenging problem in cancer chemotherapy. The combination of molecular targeted therapy and traditional chemotherapy shows potential clinical benefits. A folic acid-modified targeted drug/gene delivery system was developed to co-deliver Paclitaxel (PTX) and plasmid shBCL-2, which can enhance the sensitivity of cancer cells to chemotherapy drugs and improve therapeutic effect.
APPLIED MATERIALS TODAY
(2022)
Review
Biochemistry & Molecular Biology
Lyes Toualbi, Maria Toms, Mariya Moosajee
Summary: Inherited retinal diseases (IRDs) are a group of disorders causing progressive vision loss and affecting approximately one in 1000 people worldwide. While gene augmentation therapy using viral vectors has shown promise, non-viral gene augmentation strategies using plasmids with minimal bacterial backbones and S/MAR sequences offer potential advantages such as accommodating larger genes and reducing immune responses. Despite challenges like low retinal transfection rates, various approaches including using nanoparticles and electrical methods show promise in preclinical studies, suggesting non-viral gene therapy as a safer and effective option for future IRD treatment.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Biochemistry & Molecular Biology
Jin-Yu Liu, Xu-Ying Liu, Rui Liu, Fang Tang, Jing-Bo Yang, Quan Tang, Zhong-Lin Lu, Hai-Jun Qiao, Lan He
Summary: The combination of ANQ with hydrophilic di-(triazole-[12]aneN(3)) through different structures showed varying capabilities for gene delivery and tracing, with ANQ-A-M demonstrating strong transfection efficiency and ANQ-H-M showing poor transfection efficiency due to DNA complex aggregation and strong binding ability.
BIOORGANIC CHEMISTRY
(2021)
Article
Biochemistry & Molecular Biology
Xianqing Wang, Fatma Alshehri, Dario Manzanares, Yinghao Li, Zhonglei He, Bei Qiu, Ming Zeng, A. Sigen, Irene Lara-Saez, Wenxin Wang
Summary: To address the challenges in gene replacement strategy for RDEB, the research group utilized polymeric-based non-viral delivery systems and minicircle DNA. The improved system developed shows high potential for efficient and safe non-viral topical treatment to restore C7 in RDEB patients.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Correction
Nanoscience & Nanotechnology
Nuseibah AL Qtaish, Idoia Gallego, Alejandro J. Paredes, Ilia Villate-Beitia, Cristina Soto-Sanchez, Gema Martinez-Navarrete, Myriam Sainz-Ramos, Tania B. Lopez-Mendez, Francisco Javier Chichon, Noelia Zamarren, Eduardo Fernandez, Gustavo Puras, Jose Luis Pedraz
ACS APPLIED MATERIALS & INTERFACES
(2023)
Article
Chemistry, Multidisciplinary
Maria del Carmen Fernandez-Ramirez, Kevin Kan-Shing Ng, Margarita Menendez, Douglas V. Laurents, Ruben Hervas, Mariano Carrion-Vazquez
Summary: Understanding the early stages of amyloid formation is crucial for developing effective therapeutic strategies. Although the mechanism of tau protein aggregation, a key feature of neurodegenerative diseases, is still unclear, this study used single-molecule force spectroscopy to investigate the conformational landscape of the monomeric tau repeat domain. The researchers discovered different conformational states influenced by mutations and the chemical environment, with pathological mutations and an amyloidogenic milieu favoring expanded conformations. The findings provide insight into the complex aggregation process of tau protein and its relevance to different tauopathies.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
(2023)
Article
Biochemistry & Molecular Biology
Ivan Acebron, Maria Asuncion Campanero-Rhodes, Dolores Solis, Margarita Menendez, Carolina Garcia, M. Pilar Lillo, Jose M. Mancheno
Summary: This article introduces a new fungal lectin LBL, which can recognize and bind to various complex polysaccharide structures, and exhibit toxicity to invaders and parasites. The research found that LBL has a high affinity for insect cellular extracts, especially for fruit flies in the adult and pupa stages.
INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES
(2023)
Article
Pharmacology & Pharmacy
Nuseibah H. AL Qtaish, Ilia Villate-Beitia, Idoia Gallego, Gema Martinez-Navarrete, Cristina Soto-Sanchez, Myriam Sainz-Ramos, Tania B. Lopez-Mendez, Alejandro J. Paredes, Francisco Javier Chichon, Noelia Zamarreno, Eduardo Fernandez, Gustavo Puras, Jose Luis Pedraz
Summary: Nanodiamonds were combined with niosome to form nanodiasomes, which were evaluated for their physicochemical features, cellular internalization, cell viability, and transfection efficiency. The parameters were analyzed at different time points and temperatures. Nanodiasomes showed a stable particle size of 128 nm, except after 30 days of storage at 25 degrees C. The incorporation of nanodiamonds into niosomes resulted in a 4-fold increase in transfection efficiency, maintained over time at different temperatures. In vivo studies showed high transgene expression of nanodiasomes after subretinal and intravitreal administration in mice, both freshly prepared and after 30 days of storage at 4 degrees C.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2023)
Article
Pharmacology & Pharmacy
Fatima Garcia-Villen, Idoia Gallego, Myriam Sainz-Ramos, Jorge Ordoyo-Pascual, Sandra Ruiz-Alonso, Laura Saenz-del-Burgo, Conor O'Mahony, Jose Luis Pedraz
Summary: Drug adherence is a significant issue in medical treatment of chronic diseases like rheumatoid or psoriatic arthritis. Treated with monoclonal antibodies, the use of auto-injectors as delivery devices improves drug adherence due to their user-friendliness. This study investigated the feasibility of including monoclonal antibodies in auto-injector pens and evaluated the drug stability after concentration and freeze-drying, finding that mannitol improved stability but further investigation is required for a domain instability. These results contribute to advancing drug delivery systems for better patient experience and drug adherence.
Article
Pharmacology & Pharmacy
Jose David Celdran, Lawrence Humphreys, Desiree Gonzalez, Cristina Soto-Sanchez, Gema Martinez-Navarrete, Ivan Maldonado, Idoia Gallego, Ilia Villate-Beitia, Myriam Sainz-Ramos, Gustavo Puras, Jose Luis Pedraz, Eduardo Fernandez
Summary: Gene therapy and optogenetics show promise for treating nervous system pathologies, but viral vectors used in these approaches pose risks. Non-viral nanoparticles such as niosomes offer a potential alternative, and our study tested three niosome formulations with optogenetic plasmids in rat cortical neurons. We found successful expression of optogenetic channels, but observed changes in cell morphology and electrophysiological properties, particularly at higher concentrations of niosomes. Our results highlight the potential and the need for careful optimization in the use of niosomes for optogenetic applications.
Proceedings Paper
Geography, Physical
D. Ballesteros, A. de la Hera-Portillo, M. Llorente, R. Vila, M. M. Corral Lledo, E. Galindo Rodriguez, J. Lopez, L. Moreno, M. Menendez, P. Caldevilla, M. Ghanem, A. Hind, B. Chulli, N. E. Laftouhi, F. K. Abdel-Gawad, M. Alemparte
Summary: Karst aquifers, caves, and springs are unique sites with scientific, touristic, and water resource values. However, their management is challenging due to various factors and changes in surface hydrology and groundwater.
EUROKARST 2022: ADVANCES IN THE HYDROGEOLOGY OF KARST AND CARBONATE RESERVOIRS
(2023)
Article
Biochemistry & Molecular Biology
Giovanna Revilla, Nuseibah Al Qtaish, Pablo Caruana, Myriam Sainz-Ramos, Tania Lopez-Mendez, Francisco Rodriguez, Veronica Paez-Espinosa, Changda Li, Nuria Fucui Vallverdu, Maria Edwards, Antonio Moral, Jose Ignacio Perez, Juan Carlos Escola-Gil, Jose Luis Pedraz, Idoia Gallego, Rosa Corcoy, Maria Virtudes Cespedes, Gustavo Puras, Eugenia Mato
Summary: This study produced and assessed lenvatinib-loaded PLGA nanoparticles (NPs) as a novel therapeutic approach for aggressive thyroid tumors. The decorated NPs showed better cytotoxicity and therapeutic efficacy in vitro and in vivo compared to NPs without decoration, suggesting a promising strategy for targeted and effective administration of TKIs.
Article
Nanoscience & Nanotechnology
Daniela Rubanova, Svitlana Skoroplyas, Alena Libanska, Eva Randarova, Josef Bryja, Michaela Chorvatova, Tomas Etrych, Lukas Kubala
Summary: This study explored the use of nanocarriers to deliver dexamethasone for treating rheumatoid arthritis. The results showed that this method increased the accumulation of dexamethasone in the arthritic area, had a dose-dependent healing effect, and reduced the accumulation of the RA mediator RANKL. This study provides a feasible strategy for improving rheumatoid arthritis therapy.
NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE
(2024)
Article
Nanoscience & Nanotechnology
Thanpisit Lomphithak, Apiwit Sae-Fung, Simone Sprio, Anna Tampieri, Siriporn Jitkaew, Bengt Fadeel
Summary: In this study, iron-doped hydroxyapatite (FeHA) was evaluated as a potential nanomedicine approach for treating PDAC. The results showed that FeHA, in combination with a sublethal dose of the GPX4 inhibitor RSL3, could trigger ferroptosis in KRAS mutant PANC-1 cells while sparing normal human cells. The induction of ferroptosis by FeHA plus RSL3 was found to be dependent on the metalloreductase STEAP3 in PDAC cells.
NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE
(2024)
Review
Nanoscience & Nanotechnology
Marco Felipe Salas-Orozco, Ana Cecilia Lorenzo-Leal, Idania de Alba Montero, Nuria Patino Marin, Miguel Angel Casillas Santana, Horacio Bach
Summary: The emergence of antibiotic-resistant bacteria in severe infections is a growing concern, particularly in hospital environments. Metal-based nanomaterials have shown potential as an alternative to combat these bacteria, but there is a risk of bacterial resistance and environmental accumulation.
NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE
(2024)
