Nanotechnology-based gene delivery is the division of nanomedicine concerned with the synthesis, characterization, and functionalization of nanomaterials to be used in targeted-gene delivery applications. Nanomaterial-based gene delivery systems hold great promise for curing fatal inherited and acquired diseases, including neurological disorders, cancer, cardiovascular diseases, and acquired immunodeficiency syndrome (AIDS). However, their use in clinical applications is still controversial. To date, the Food and Drug Administration (FDA) has not approved any gene delivery system because of the unknown long-term toxicity and the low gene transfection efficiency of nanomaterials in vivo. Compared to viral vectors, nonviral gene delivery vectors are characterized by a low preexisting immunogenicity, which is important for preventing a severe immune response. In addition, nonviral vectors provide higher loading capacity and ease of fabrication. For these reasons, this review article focuses on applications of nonviral gene delivery systems, including those based on lipids, polymers, graphene, and other inorganic nanoparticles, and discusses recent advances in nanomaterials for gene therapy. Methods of synthesizing these nanomaterials are briefly described from a materials science perspective. Also, challenges, critical issues, and concerns about the in vivo applications of nanomaterial-based gene delivery systems are discussed. It should be noted that this article is not a comprehensive review of the literature.
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http://dx.doi.org/10.1039/c6bm00441e | DOI Listing |
Sci Rep
January 2025
Department of Burn and Plastic Surgery, Affiliated Hospital of Nantong University, Nantong, 226001, China.
Hepatocyte growth factor (HGF) is a substance that stimulates the proliferation of hepatocytes which promote healing. We developed a macrophage membrane-encapsulated nanosphere drug delivery system containing HGF for the study of burn wound healing. Twenty-seven Sprague-Dawley rats were randomly divided into three groups: a saline control (NS) group, an engineered macrophage membrane-encapsulated nanospheres (ETMM@NPS) group, and an engineered macrophage membrane-encapsulated nanospheres treatment with HGF-loaded gene (HGF@ETMM@NPS) group.
View Article and Find Full Text PDFCancer Gene Ther
January 2025
Neurophysiology Unit, Cardiac Electrophysiology Research and Training Center, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand.
Ovarian cancer is a leading cause of cancer-related deaths in women, and the development of chemoresistance remains a major challenge during and after its treatment. Exosomes, small extracellular vesicles involved in intercellular communication, have emerged as potential biomarkers and therapeutic targets in ovarian cancer. This review summarizes the current literature on differences in exosomal protein/gene expression between chemosensitive and chemoresistant ovarian cancer, and the effects of exosomal modifications on chemotherapeutic response.
View Article and Find Full Text PDFNature
January 2025
Genomic Medicine Center, Children's Mercy Kansas City, Kansas City, MO, USA.
Personalized antisense oligonucleotides (ASOs) have achieved positive results in the treatment of rare genetic disease. As clinical sequencing technologies continue to advance, the ability to identify patients with rare disease harbouring pathogenic genetic variants amenable to this therapeutic strategy will probably improve. Here we describe a scalable platform for generating patient-derived cellular models and demonstrate that these personalized models can be used for preclinical evaluation of patient-specific ASOs.
View Article and Find Full Text PDFCarbohydr Polym
March 2025
Department of Biomedical Engineering, College of Design and Engineering, National University of Singapore, 15 Kent Ridge Crescent, Singapore 119276, Singapore; National University of Singapore (Suzhou) Research Institute, Suzhou, Jiangsu 215123, China; National University of Singapore (Chongqing) Research Institute, Yubei, Chongqing 401120, China; NUS Environmental Research Institute (NERI), National University of Singapore, 5A Engineering Drive 1, Singapore 117411, Singapore. Electronic address:
The combination of chemotherapy and gene therapy holds promise in treating cancer. A key strategy is to use small interfering RNAs (siRNAs) to silence programmed death-ligand 1 (PD-L1) expression in cancer cells, disrupting tumor immune evasion and enhancing anticancer treatments, particularly when used in conjunction with chemotherapy drugs such as doxorubicin (Dox). However, effective codelivery of drugs and genes requires carefully designed carriers and complex synthesis procedures.
View Article and Find Full Text PDFCarbohydr Polym
March 2025
Department of Pharmacy, The Fourth Affiliated Hospital of China Medical University, Shenyang 110032, China. Electronic address:
Cyclodextrins (CDs) are cyclic polysaccharides characterized by their unique hollow structure, making them highly effective carriers for pharmaceutical agents. CD-based delivery systems are extensively utilized to enhance drug stability, increase solubility, improve oral bioavailability, and facilitate controlled release and targeted delivery. This review initially provides a concise overview of nano drug delivery systems, followed by a detailed introduction of the structural features and benefits of CDs.
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