Article
Medicine, Research & Experimental
Cynthia D. Anderson, Jennifer Ataam Arthur, Yuan Zhang, Nike Bharucha, Ioannis Karakikes, Ralph V. Shohet
Summary: CRISPR-Cas9-based genome editing technologies have the potential for clinical translation, but delivering nucleic acids into target cells in vivo is challenging. This study presents a new method using focused ultrasound targeted microbubble destruction to deliver CRISPR-Cas9 base editing vectors to the mouse liver. The results demonstrate successful base editing in mouse liver cells, but with lower specificity and more off-target base exchange in vivo.
MOLECULAR THERAPY NUCLEIC ACIDS
(2023)
Review
Pharmacology & Pharmacy
Jieting Wang, Luying Yu, Ao Zhou, Jie Liu, Kai Wang, Ying Luo, Fang Wang
Summary: Cardiovascular diseases are a leading cause of morbidity and mortality worldwide, with gene therapy offering new possibilities for treatment. Non-viral gene therapies are gaining attention for their safety and ease of production, providing new insights for promoting cardiac repair/regeneration.
Article
Pharmacology & Pharmacy
Asma Ghaemi, Masoume Vakili-Azghandi, Khalil Abnous, Seyed Mohammad Taghdisi, Mohammad Ramezani, Mona Alibolandi
Summary: Gene therapy has the potential to prevent or treat various genetic or acquired diseases by regulating gene and protein expression. Effective delivery systems are needed to ensure successful gene therapy, and oral delivery approaches show promise in treating diseases such as inflammatory bowel disease and cancer. This review provides an update on the development of oral gene delivery techniques for gene therapy and vaccination purposes.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2023)
Review
Biotechnology & Applied Microbiology
Sepideh Jahangiri, Maedeh Rahimnejad, Narges Nasrollahi Boroujeni, Zarrin Ahmadi, Puria Motamed Fath, Sepideh Ahmadi, Moein Safarkhani, Navid Rabiee
Summary: The overall success in launching discovered drugs is restricted by the high rate of late-stage failures. Reliable methods are needed to predict the effectiveness and toxicity of medicines early in the drug development process. Modern in vitro disease models and advanced technology, such as 3D bioprinting, can be used to expedite drug discovery. 3D bioprinting has the potential to revolutionize pharmaceutical studies and improve gene delivery and tissue engineering.
JOURNAL OF GENE MEDICINE
(2022)
Review
Biotechnology & Applied Microbiology
Chenfei Wang, Chaolan Pan, Haiyang Yong, Feifei Wang, Tao Bo, Yitong Zhao, Bin Ma, Wei He, Ming Li
Summary: Gene therapy holds great promise for treating a wide range of genetic diseases by delivering functional genes into targeted cells or tissues. However, the lack of safe and efficient gene delivery vehicles remains a major obstacle to its clinical implementation. This review comprehensively outlines the novel non-viral gene delivery vectors with potential applications in gene therapy.
JOURNAL OF NANOBIOTECHNOLOGY
(2023)
Review
Chemistry, Multidisciplinary
Shuai Qu, Renfa Liu, Nisi Zhang, Yunxue Xu, Xiuli Yue, Zhifei Dai
Summary: This article reviews the recent advances in targeted non-viral nucleic acid delivery for gene therapy of atherosclerosis, including hepatocyte-targeted delivery and delivery to different cells within the plaques.
Article
Biochemistry & Molecular Biology
Daniel Keim, Katrin Gollner, Ulrich Gollner, Valerie Jerome, Ruth Freitag
Summary: The study demonstrated that a 24-armed PDMAEMA nano-star can achieve high levels of transgene expression (40%) in human B cells while maintaining sufficient cell viability. Optimal transfection results were obtained with four days of stimulation with rhCD40L post-thawing. Furthermore, B cell subsets were found to impact transfection outcomes, highlighting the importance of considering the complexity and heterogeneity of B cell populations.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Biochemical Research Methods
Raghu Ramanathan, Nathan A. Delvaux, Kevin G. Rice
Summary: We developed an improved in vitro transfection assay for testing the efficiency of non-viral vector DNA nanoparticle transfection of primary hepatocytes. The assay was optimized for cell plating number, DNA dose, and PEI to DNA ratio, and provides reliable in vitro expression results for direct comparison of different non-viral gene delivery vectors.
ANALYTICAL BIOCHEMISTRY
(2022)
Article
Engineering, Environmental
Xuefeng Tang, Zhao Wang, Ying Zhang, Wei Mu, Xiaojun Han
Summary: This review summarizes the delivery modes of the CRISPR-Cas9 system and recent progress in delivering it using non-viral nanocarriers. Various types of nanocarriers, including lipid-based, protein-based, polymer-based, gold-based, and zeolitic imidazolate framework-based carriers, are discussed in terms of their design and gene editing efficiency. The challenges and future prospects of CRISPR-Cas9 non-viral nanocarriers are also proposed.
CHEMICAL ENGINEERING JOURNAL
(2022)
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)
Article
Biophysics
Yan Wang, Feiyang Liu, Chunxiao Pu, Zhangfa Tong, Mian Wang, Jianyi Wang
Summary: In this study, novel dual-targeting fluorescent probes were developed by modifying imidazole and galactose. These probes showed high selectivity and sensitivity towards Fe3+ and exhibited excellent hepatic targeting capacity and lysosome-targeting capacities. They have the potential to be used for precise diagnosis and treatment of liver lysosomal iron-related diseases.
BIOSENSORS & BIOELECTRONICS
(2022)
Article
Engineering, Biomedical
Guo Chen, Shaohui Deng, Mingxiang Zuo, Jin Wang, Du Cheng, Bin Chen
Summary: By using the nonviral CRISPRa system, the expressions of TGF-81 and VEGF-A genes were activated to enhance osteogenesis in MC3T3-E1 cells. The engineered cells implanted with a tunable dual-crosslinked hydrogel also promoted the healing of mice calvarial bone defects.
MATERIALS TODAY BIO
(2022)
Review
Pharmacology & Pharmacy
Qi Shuai, Fangtao Zhu, Mengdan Zhao, Yunfeng Yan
Summary: The article mainly discusses the challenges faced in mRNA-based therapeutics and the insufficient requirements for delivery vehicles, while emphasizing the advancements in mRNA delivery.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2021)
Article
Polymer Science
Mehmet Koray Gok, Kamber Demir, Erdal Cevher, Serhat Pabuccuoglu, Saadet Ozgumus
Summary: This study successfully synthesized branched poly(beta-aminoester) (PBAE) with high transfection efficiency and prepared PBAE nanoparticles using two different methods. In vitro studies on primary ovine fibroblast cells showed that the formulated PBAE had a high transfection efficiency, exhibiting almost 1.5 times more efficiency than the commercial product Lipofectamine.
EUROPEAN POLYMER JOURNAL
(2022)
Article
Engineering, Biomedical
Meirong Li, Zheng-Ian Lin, Jingyu Yang, Haoqiang Huang, Guan-Lin Liu, Qiqi Liu, Xinmeng Zhang, Ying Zhang, Zhourui Xu, Haoming Lin, Yujuan Chai, Xin Chen, Bao-Tsan Ko, Jia Liu, Chih-Kuang Chen, Chengbin Yang
Summary: Osteosarcoma is a highly invasive and deadly cancer that often occurs in children and adolescents. The overexpression of PLK1 gene in tumors promotes cancer cell proliferation and transformation, making it a therapeutic target for osteosarcoma. However, current RNA interference-based therapies lack a safe and efficient nonviral gene vector. In this study, biodegradable and CO2-derivative cationic poly(vinylcyclohexene carbonates) (CPCHCs) were used as gene vectors for siPLK1 therapeutic strategy in osteosarcoma treatment. Among them, CPCHC60 showed excellent performance in gene transfection efficiency, endo-lysosome escaping, biodegradability, and biosafety. The treatment with CPCHCs/siRNA nanoparticles significantly down-regulated the expression level of PLK1 gene in osteosarcoma cells, leading to cell arrest and apoptosis, resulting in significant tumor regression both in vitro and in vivo. This study provides new insights into the development of superior nonviral gene vectors for cancer treatment and holds high potential for translational nanomedicine applications.
ADVANCED HEALTHCARE MATERIALS
(2023)
Article
Pharmacology & Pharmacy
Kevin Matha, Giovanna Lollo, Giuseppe Taurino, Renaud Respaud, Ilaria Marigo, Molood Shariati, Ovidio Bussolati, An Vermeulen, Katrien Remaut, Jean-Pierre Benoit
EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS
(2020)
Article
Nanoscience & Nanotechnology
Molood Shariati, Giovanna Lollo, Kevin Matha, Benedicte Descamps, Christian Vanhove, Leen Van de Sande, Wouter Willaert, Lieve Balcaen, Frank Vanhaecke, Jean-Pierre Benoit, Wim Ceelen, Stefaan C. De Smedt, Katrien Remaut
ACS APPLIED MATERIALS & INTERFACES
(2020)
Review
Pharmacology & Pharmacy
Kevin Matha, Brice Calvignac, Jean-Pierre Gangneux, Jean-Pierre Benoit
Summary: Visceral leishmaniasis is a neglected disease in many countries, but nanomedicine has shown success in treating it, particularly with liposomal amphotericin B's remarkable cure rates. However, developing more efficient strategies like targeting functionalization, oral formulations, or combined therapies face numerous obstacles and raise many questions.
EXPERT OPINION ON DRUG DELIVERY
(2021)
Article
Oncology
Anne Bonhoure, Laurent Henry, Marie Morille, Nesrine Aissaoui, Gaetan Bellot, Pierre-Emmanuel Stoebner, Michel Vidal
Summary: Cutaneous melanoma is the deadliest form of skin cancer and early detection is crucial. Exosomal melanotransferrin has been identified as a potential biomarker for melanoma, showing high levels in melanoma patients compared to noncancer patients and being able to be detected through an ELISA method.
Review
Pharmacology & Pharmacy
Sarah Le Saux, Anne Aubert-Pouessel, Khaled Elhady Mohamed, Pierre Martineau, Laurence Guglielmi, Jean-Marie Devoisselle, Philippe Legrand, Joel Chopineau, Marie Morille
Summary: Protein therapeutics offer higher specificity and lower toxicity compared to chemicals, with potential for broad application. Delivery systems are necessary for maximizing their biological potential, and extracellular vesicles (EVs) as natural protein nanocarriers show great promise.
ADVANCED DRUG DELIVERY REVIEWS
(2021)
Article
Biology
Jihad Karam, Julie Constanzo, Alexandre Pichard, Laurent Gros, Joel Chopineau, Marie Morille, Jean-Pierre Pouget
Summary: This study investigated the effects of sEVs produced by irradiated cells on bystander cytotoxic effects and therapeutic efficacy. In vitro experiments showed that sEVs had cytotoxic effects on recipient cells. However, no systemic effects were observed in vivo.
INTERNATIONAL JOURNAL OF RADIATION BIOLOGY
(2023)
Review
Pharmacology & Pharmacy
Amanda K. A. Silva, Marie Morille, Max Piffoux, Surendar Arumugam, Phlippe Mauduit, Jerome Larghero, Arnaud Bianchi, Kelly Aubertin, Olivier Blanc-Brude, Daniele Noel, Emilie Velot, Celia Ravel, Celine Elie-Caille, Anna Sebbagh, Chantal Boulanger, Claire Wilhelm, Gabriel Rahmi, Isabelle Raymond-Letron, Kondareddy Cherukula, Tristan Montier, Christophe Martinaud, Jean-Marie Bach, Olivier Favre-Bulle, Jolanda Spadavecchia, Christian Jorgensen, Philippe Menasche, Clotilde Aussel, Joel Chopineau, Mathilde Mosser, Matti Ullah, Nicolas Sailliet, Nathalie Luciani, Noelle Mathieu, Pierre-Emmanuel Rautou, Sophie Brouard, Wilfrid Boireau, Sebastien Jauliac, Marianne Dedier, Jean-Hugues Trouvin, Florence Gazeau, Marina Trouillas, Juliette Peltzer, Antoine Monsel, Sebastien Banzet
Summary: The translation of EV research into medicinal products presents an exciting and challenging perspective, requiring fulfillment of quality, safety, and efficacy criteria. Active contributions from regulatory agencies, expert networks, and the newly established EVOLVE group in France play important roles in this translation process.
ADVANCED DRUG DELIVERY REVIEWS
(2021)
Correction
Chemistry, Medicinal
Lotfi Boudjema, Hanna Aarrass, Marwa Assaf, Marie Morille, Gaelle Martin-Gassin, Pierre-Marie Gassin
JOURNAL OF CHEMICAL INFORMATION AND MODELING
(2021)
Article
Engineering, Biomedical
Jeremy Salvador, Jade Berthelot, Claire Bony, Baptiste Robin, Josephine Lai Kee Him, Daniele Noel, Emmanuel Belamie, Marie Morille
Summary: This study introduces a new siRNA nanovector called SELF, which has a tunable size and high transfection efficiency for primary human mesenchymal stromal cells (hMSC). The study demonstrates that the loading capacity and release kinetics of SELF can be controlled by associating it with porous 3D collagen microspheres. The different release profiles of siRNA from the microspheres result in different transfection kinetics of hMSCs, highlighting the versatility of this gene-activated matrix for regenerative medicine applications.
ACTA BIOMATERIALIA
(2022)
Article
Pharmacology & Pharmacy
Francois Noverraz, Baptiste Robin, Solene Passemard, Benedicte Fauvel, Jessy Presumey, Emilie Rigal, Alan Cookson, Joel Chopineau, Pierre Martineau, Martin Villalba, Christian Jorgensen, Anne Aubert-Pouessel, Marie Morille, Sandrine Gerber-Lemaire
Summary: The newly developed excipient C16TreSuc can stabilize antibodies and enable pulmonary immunotherapy for the treatment of COVID-19 and other lung diseases.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2023)
Article
Pharmacology & Pharmacy
L. Simon, A. Coeurvolan, M. Colpaert, J. M. Devoisselle, M. Morille, N. Marcotte, V. Lapinte
Summary: This study evaluated the potential of lipopolyoxazolines, which are amphiphilic polyoxazolines bearing lipid chains, for efficient intracellular delivery. Different lipid chains were associated with poly(2-methyl-2-oxazoline) block, and their physicochemical characteristics and impact on cell viability and internalization capacity were assessed. The results showed that the linear saturated lipid chain had the highest cell internalization and good cell viability. Comparison with PEG reference (DSPE-PEG) liposomes revealed that lipopolyoxazoline liposomes exhibited significantly improved intracellular delivery, highlighting the difficulty of PEGylated liposomes to enter cells by endocytosis. This study promotes the value of lipopoly(oxazoline) as an alternative to lipopoly(ethylene glycol) for effective intracellular delivery.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2023)
Review
Cell Biology
Laurianne Simon, Vincent Lapinte, Marie Morille
Summary: Extracellular vesicles (EVs) are key mediators of intercellular communication and have great potential in various fields. However, their complexity and biological origin pose challenges in their identification and therapeutic use. This review highlights the importance of multidisciplinary research involving polymers to address these limitations.
JOURNAL OF EXTRACELLULAR VESICLES
(2023)
Review
Pharmacology & Pharmacy
Sarah Le Saux, Anne Aubert-Pouessel, Lyria Ouchait, Khaled Elhady Mohamed, Pierre Martineau, Laurence Guglielmi, Jean-Marie Devoisselle, Philippe Legrand, Joel Chopineau, Marie Morille
Summary: This article highlights the importance of proteins as drugs, discusses the significance of intracellular targeting therapy, explores the challenges faced by therapeutic proteins, and reviews the latest advancements in delivery system research.
ADVANCED THERAPEUTICS
(2021)
Article
Chemistry, Physical
Ayman El Jundi, Marie Morille, Nadir Bettache, Audrey Bethry, Jade Berthelot, Jeremy Salvador, Sylvie Hunger, Youssef Bakkour, Emmanuel Belamie, Benjamin Nottelet
JOURNAL OF COLLOID AND INTERFACE SCIENCE
(2020)
Meeting Abstract
Endocrinology & Metabolism
J. Laget, F. Galtier, A. Nouvel, M. Morille, P. Geraud, D. Nocca, N. Builles, S. Rebuffat, A. Lajoix
Article
Pharmacology & Pharmacy
Greta Camilla Magnano, Marika Quadri, Elisabetta Palazzo, Roberta Lotti, Francesca Loschi, Stefano Dall'Acqua, Michela Abrami, Francesca Larese Filon, Alessandra Marconi, Dritan Hasa
Summary: This study aimed to investigate the loading of sildenafil citrate in three commercial transdermal vehicles using 3D full-thickness skin equivalent and compare the results with permeability experiments using porcine skin. The results showed that the results obtained using the 3D skin equivalent were comparable to those obtained using porcine skin, suggesting that the 3D skin model can be a valid alternative for ex-vivo skin absorption experiments.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2024)
Article
Pharmacology & Pharmacy
James W. Mckeage, Andrew Z. H. Tan, Andrew J. Taberner
Summary: Needle-free jet injection is a promising alternative drug delivery technique that offers rapid, non-invasive, and large-volume injections. The study presents a prototype multi-orifice nozzle and a computational fluid dynamic model to demonstrate the feasibility and effectiveness of this technology.
INTERNATIONAL JOURNAL OF PHARMACEUTICS
(2024)