Review
Genetics & Heredity
Muhammad Hammad Butt, Muhammad Zaman, Abrar Ahmad, Rahima Khan, Tauqeer Hussain Mallhi, Mohammad Mehedi Hasan, Yusra Habib Khan, Sara Hafeez, Ehab El Sayed Massoud, Md Habibur Rahman, Simona Cavalu
Summary: Gene therapy has gained significant importance in medical research as a potential treatment strategy for various diseases. The delivery of genes to target cells inside the body requires suitable vectors to protect the gene cargo and enable efficient delivery. Viral vectors and nonviral vectors have been developed to serve as carriers for gene delivery, with viral vectors being highly efficient and nonviral vectors providing sustainable gene expression with minimal side effects.
Article
Engineering, Biomedical
Guoxin Tan, Dandan Liu, Renfang Zhu, Hao Pan, Jiayang Li, Weisan Pan
Summary: This study developed a core-shell nanoplatform for targeted delivery of genes to the retina, showing potential for treating retinal diseases such as AMD. The platform demonstrated safety and efficiency in delivering genes to the retina, inhibiting VEGF expression and angiogenesis. This nanoplatform has the capability to overcome barriers in gene delivery to the retina, offering a promising approach for retinal gene therapy.
ACTA BIOMATERIALIA
(2021)
Article
Chemistry, Multidisciplinary
Jiaojiao Zhang, Zixuan Wang, Jiwei Min, Xuelin Zhang, Rongxin Su, Yuefei Wang, Wei Qi
Summary: A transfection formulation is developed using an auxiliary lipid (DOTAP) added to the peptide, resulting in a transfection efficiency of 72.6% for pDNA, which is comparable to Lipofectamine 2000. The designed KHL peptide-DOTAP complex exhibits good biocompatibility and significantly improves mRNA delivery compared to KHL or DOTAP alone. The complex also achieves efficient endolysosomal escape, providing a new platform for enhancing peptide vector transfection efficiency.
Review
Medicine, Research & Experimental
Sanjay Arora, Divya Sharma, Buddhadev Layek, Jagdish Singh
Summary: Alzheimer's disease is a progressive and incurable CNS disorder characterized by the accumulation of A beta plaques and NFTs, as well as synaptic loss and neuronal signaling imbalance. Current treatments aim to manage symptoms and maintain cognitive functions while slowing down memory loss. Nonviral gene therapy shows promise in addressing these challenges, with advancements in technology offering potential solutions in clinical trials.
MOLECULAR PHARMACEUTICS
(2021)
Review
Chemistry, Multidisciplinary
Hengde Li, Shuai Zha, Haolan Li, Haito Liu, Ka-Leung Wong, Angelo H. All
Summary: Dendrimers are polymers with well-defined 3D branched structures that are widely used in neurotheranostics and biomedical applications, particularly as nanocarrier vectors. They can improve the accuracy of diagnostic imaging processes by loading imaging agents. Combining pharmaceutical agents and anticancer drugs with dendrimers enhances their solubility and efficiency, and modifying ligands on dendrimers' surface can construct effective targeted delivery systems.
Article
Multidisciplinary Sciences
Jiaxuan Chen, Dandan Zhu, Baoping Lian, Kangjie Shi, Peng Chen, Ying Li, Wenyi Lin, Ling Ding, Qiulin Long, Yang Wang, Erik Laurini, Wenjun Lan, Yun Li, Aura Tintaru, Caoyun Ju, Can Zhang, Sabrina Pricl, Juan Iovanna, Xiaoxuan Liu, Ling Peng
Summary: Nucleic acid therapeutics serve as an important drug modality for targeting undruggable targets, responding to evolving pathogens, and precision medicine at the gene level. Dendrimers, as precision delivery systems, have been utilized for selective and on-demand delivery of DNA and siRNA. The properties and performance of these dendrimers were systematically studied, and they exhibited cargo-adaptive and selective delivery, tumor targeting, and redox-responsive cargo release. The dendrimers outperformed currently available vectors in tumor and cancer cell-specific delivery of siRNA and DNA therapeutics in various cancer models. This study provides a pathway for engineering tailor-made vectors for nucleic acid delivery and precision medicine.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2023)
Article
Biotechnology & Applied Microbiology
Rkia El-Kharrag, Kurt E. Berckmueller, Ravishankar Madhu, Margaret Cui, Gabriela Campoy, Heather M. Mack, Carl B. Wolf, Anai M. Perez, Olivier Humbert, Hans-Peter Kiem, Stefan Radtke
Summary: This study evaluated the feasibility of polymer-based nanoparticles (NPs) for the delivery of mRNA and nucleases to human CD34+ cells. The results showed that NP-mediated gene editing had high efficiency and low toxicity, with a reduced amount of reagents compared to electroporation. Additionally, the use of NPs enhanced the engraftment potential of HSCs in a mouse xenograft model.
Article
Multidisciplinary Sciences
Shubha R. L. Malla, Archana Gujjari, Carlos E. Corona, Gary W. Beall, L. Kevin Lewis
Summary: This study investigates the spectrophotometric and sedimentation properties of two clays in aqueous solutions and their abilities to bind with small and large nucleic acids. The results show that HNT has strong binding capabilities for small nucleic acids, while kaolinite only binds efficiently with small nucleic acids in the presence of Mg2+ ions. Large linear double-stranded DNAs and circular plasmid DNAs have poor binding with kaolinite but can strongly associate with HNT. These findings demonstrate that the interactions between clays and nucleic acids depend on the type and conformation of each nucleic acid.
Article
Nanoscience & Nanotechnology
Rong Ni, Ruilu Feng, Ying Chau
Summary: Nonviral vectors are a safer alternative to viral vectors for gene therapy, and artificial viruses combining viral and nonviral vector strengths have emerged as safe and efficient gene delivery vectors. In this study, peptide-DNA coassembled nanoparticles were developed as a new efficient vector for gene delivery, achieving high transfection efficiency through the introduction of rationally designed functional short peptide modules. These nanoparticles achieved over 90% transfection efficiency in a serum-containing medium. This work demonstrates the effectiveness of the module-based design approach and lays the groundwork for potential biomedical applications of peptide-based biomaterials.
ACS APPLIED NANO MATERIALS
(2023)
Article
Chemistry, Multidisciplinary
Nasha Qiu, Guowei Wang, Jinqiang Wang, Quan Zhou, Mengyu Guo, Yaling Wang, Xuhao Hu, Huige Zhou, Ru Bai, Min You, Zhen Zhang, Chunying Chen, Ying Liu, Youqing Shen
Summary: A novel IL12 gene-delivery vector was designed to efficiently transfect both cancer cells and TAMs, turning them into a factory for IL12 production, which effectively activates anticancer immune responses and remodels the tumor microenvironment. The intravenously administered vector slows tumor growth and doubles survival in three animal models with minimal systemic toxicities.
ADVANCED MATERIALS
(2021)
Review
Engineering, Biomedical
Heng Zhou, Yan He, Wei Xiong, Shuili Jing, Xingxiang Duan, Zhiyun Huang, Gurek S. Nahal, Youjian Peng, Mingchang Li, Yaoqi Zhu, Qingsong Ye
Summary: Mesenchymal stem cells (MSCs) have promising potential for neural regeneration therapy due to their plasticity and accessibility. However, the therapeutic efficacy of MSCs alone is often inadequate, leading to the use of genetic engineering to enhance their abilities. Different methods of gene transfer have specific advantages and disadvantages. MSCs can also be modified to improve their neural repair abilities. This review focuses on gene transport technologies for engineering MSCs and the selection of optimal delivery genes, as well as the prospects and challenges of their application in animal models of neurological diseases.
BIOACTIVE MATERIALS
(2023)
Review
Chemistry, Multidisciplinary
Peng Yang, Athena Yue-Tung Lee, Jingjing Xue, Shih-Jie Chou, Calvin Lee, Patrick Tseng, Tiffany X. Zhang, Yazhen Zhu, Junseok Lee, Shih-Hwa Chiou, Hsian-Rong Tseng
Summary: The CRISPR/Cas9 genome editing system has revolutionized the field of therapeutic applications for genetic diseases and cancers. Efficient delivery of the system in vivo remains a significant challenge. Non-viral nano-vectors offer advantages over viral vectors and can be used to deliver CRISPR/Cas9 cargoes for targeted gene editing. The article highlights recent advances in nano-vector delivery for treating cancers and genetic diseases and proposes strategies for future development.
Article
Chemistry, Multidisciplinary
Guowei Wang, Siqin Chen, Nasha Qiu, Bihan Wu, Dingcheng Zhu, Zhuxian Zhou, Ying Piao, Jianbin Tang, Youqing Shen
Summary: The virus-mimicking polymeric vector presented can efficiently transfect genes by sticking onto the cell membrane and ejecting DNA into the cytoplasm, bypassing endo-/lysosomal DNA degradation and achieving high gene expression efficiency.
Article
Nanoscience & Nanotechnology
Yaqi Cao, Junshi Zhang, Lan Wang, Mingjun Cen, Wenchao Peng, Yang Li, Fengbao Zhang, Juan Tan, Xiaobin Fan
Summary: Gene therapy is a revolutionary biomedical technique that requires safe and efficient intracellular gene delivery vectors for successful treatment. A recent study introduces dual-functionalized nanosheets as nonviral vectors, with superior gene transfection performance and lower cytotoxicity, especially at low dosages.
ACS APPLIED NANO MATERIALS
(2021)
Article
Instruments & Instrumentation
Frazer Sinclair, Anjuman A. Begum, Charles C. Dai, Istvan Toth, Peter M. Moyle
Summary: The CRISPR/Cas9 genome editing system is a groundbreaking technology that has revolutionized genome editing for therapeutic, diagnostic, and precision medicine applications. The safe and efficient delivery of the CRISPR/Cas9 system has been a critical challenge. Viral vectors have shown promise, but face limitations for clinical applications. Nonviral delivery vectors, such as lipid, polymer, peptides, and inorganic nanoparticle-based systems, have emerged as viable alternatives. This review discusses the molecular mechanisms of CRISPR/Cas9, current delivery strategies, approaches for reducing off-target editing, and future directions for clinical trials.
DRUG DELIVERY AND TRANSLATIONAL RESEARCH
(2023)
