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Polysaccharide-Based Delivery Systems for CRISPR/Cas Gene Therapy: Overcoming Challenges and Advancing Pharmaceutical Solutions
Authors: Deeksha Manchanda, Sunil Kumar and Manish MakhijaAvailable online: 10 March 2025More LessThe advent of CRISPR/Cas gene-editing technology has revolutionized molecular biology, offering unprecedented precision and potential in treating genetic disorders, cancers, and other complex diseases. However, for CRISPR/Cas to be truly effective in clinical settings, one of the most significant challenges lies in the delivery of the CRISPR components, including guide RNA (gRNA) and Cas protein, into specific cells or tis Read More
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The Antimicrobial Peptide Pipeline: A Bacteria-Centric AMP Predictor
Authors: Werner Pieter Veldsman, Qi Zhang, Qian Zhao, Lu Zhang and Zou YuanjieAvailable online: 26 February 2025More LessIntroduction Antimicrobial peptides (AMPs), unlike antibiotics, are encoded in genomes. AMPs are exported from the cell after expression and translation. In the case of bacteria, the exported peptides target other microbes to give the producing bacterium a competitive edge. While AMPs are sought after for their similar antimicrobial activity to traditional antibiotics, it is difficult to predict which combinations of amino acids Read More
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Gene Therapy and Gene Editing: Current Trends and Future Prospects of Molecular Medicine
Available online: 18 February 2025More LessGene therapy and genome editing have emerged as transformative approaches in the management of a diverse range of genetic and acquired diseases. This evaluation offers a thorough examination of the present state and prospects of these innovative technologies. Gene therapy is a prospective approach to the treatment and prevention of a variety of conditions, including complex cancers and inherited genetic disord Read More
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Gene Therapy: Transforming the Battle Against Pancreatic Cancer
Authors: Rohit Sharma, Sourabh Kumar, Rashmi Ghosh, Kumari Komal and Manish KumarAvailable online: 06 February 2025More LessPancreatic cancer remains one of the most aggressive and lethal malignancies, with a dismal prognosis despite advancements in conventional treatment modalities. Gene therapy has emerged as a promising approach to combat pancreatic cancer by targeting the underlying genetic alterations and harnessing the power of the immune system. This review explores the current landscape of gene therapy strategies for Read More
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Targeting Undruggable Proteins: The siRNA Revolution Beyond Small Molecules - Advances, Challenges, and Future Prospects in Therapeutic Innovation
Available online: 04 February 2025More LessThe field of drug discovery has long been challenged by the existence of “undruggable” proteins - targets that have resisted traditional small molecule approaches due to their structural or functional characteristics. This review explores the revolutionary potential of small interfering RNA (siRNA) technology in addressing these elusive targets, marking a paradigm shift in therapeutic development. We discuss the historical d Read More
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Molecular Insights into RNA Modifications and their Role in Shaping Immune Responses and Tumor Microenvironments
Authors: Ashiq Ali, Tehreem Ajmal, Aisha Khatoon, Kaynaat Akbar, Urooj Irshad, Bilal Murtaza, Ziyi Ji, Abdullah Ali, Qaisar Tanveer and Zhongjing SuAvailable online: 21 January 2025More LessRNA modifications play crucial roles in immune system development and function, with dynamic changes essential for diverse cellular processes. Innovative profiling technologies are invaluable for understanding the significance of these modifications in immune cells, both in healthy and diseased states. This review explores the utility of such technologies in uncovering the functions of RNA modifications and their impact o Read More
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Development of Lentiviral Packaging Cells and Scale Up of Production to Meet the Growing Demand in Cell and Gene Therapy
Authors: Yasemin van Heuvel and Jörn StitzAvailable online: 13 January 2025More LessGamma-Retroviral (RVVs) and lentiviral vectors (LVVs) represent indispensable tools in somatic gene therapy, mediating the efficient, stable transfer of therapeutic genes into a variety of human target cells. LVVs, in contrast to RVVs, are capable of stably genetically modifying non-proliferating target cells, making them the superior instrument in cell and gene therapy. To date, the LVV manufacturing process employ Read More
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O-substituted Tertiary Amine-chitosans as Promising Nanocarriers for siRNA Delivery
Available online: 10 January 2025More LessIntroduction The clinical translation of chitosan-based formulations for siRNA delivery has been partially limited by their poor stability/solubility at physiological conditions, although they have good biocompatibility and cost-effectiveness. Method In this study, the chitosan was O-substituted with diisopropylethylamine (DIPEA) groups, which improved its solubility at pH 7.4 by increasing the degree of ionization and enhanced the ab Read More
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Implications of EBV-Encoded and EBV-Related miRNAs in Tumors
Authors: Prankur Awasthi, Arjun Singh Kohli, Manish Dwivedi and Saba HasanAvailable online: 08 January 2025More LessOver 90% of people are infected with the human g-herpesvirus known as the Epstein-Barr virus (EBV). Cancers, such as gastric carcinoma, non-Hodgkin’s lymphoma, nasopharyngeal carcinoma, Hodgkin’s lymphoma, and Burkitt lymphoma, are thought to be linked with EBV. It is noteworthy that the first virus discovered that encodes microRNAs (miRNAs) was EBV, and these miRNAs show expression at the different phases of Read More
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Plasmalogens Activate AKT/mTOR Signaling to Attenuate Reactive Oxygen Species Production in Spinal Cord Injury
Authors: Mengdan Cheng, Yan Gao, Yiqing Wu, Liangliang Zhang, Bai Xu and Xiaojie LuAvailable online: 07 January 2025More LessBackground Plasmalogens, the primary phospholipids in the brain, possess intrinsic antioxidant properties and are crucial components of the myelin sheath surrounding neuronal axons. While their neuroprotective effects have been demonstrated in Alzheimer's disease, their potential benefits in spinal cord injury remain unexplored. This study investigates the reparative effects of plasmalogens on spinal cord injury and the underl Read More
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CRISPR/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases as Therapeutic Delivery; Current Applications and Challenges
Authors: Fatemeh Saberi, Zeinab Yousefi-Najafabadi, Forough Shams, Zeinab Dehghan, Sepideh Ahmadi, Tayyebeh Pilehchi, Effat Noori, Zeinab Esmaeelzadeh, Maryam Bazgiri, Rezvan Mohammadi, Farzaneh Khani, Marzieh Sameni, Parisa Moradbeigi, Gholam Ali Kardar, Mohammad Salehi, Yong Teng and Vahid JajarmiAvailable online: 07 January 2025More LessThe 5,000 to 8,000 monogenic diseases are inherited disorders leading to mutations in a single gene. These diseases usually appear in childhood and sometimes lead to morbidity or premature death. Although treatments for such diseases exist, gene therapy is considered an effective and targeted method and has been used in clinics for monogenic diseases since 1989. Monogenic diseases are good candidates for novel th Read More
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The Role of Immunotherapy in Lung Cancer Treatment: Current Strategies, Future Directions, and Insights into Metastasis and Immune Microenvironment
Authors: Chen Lu Li, Xin Yuan and Ping YiAvailable online: 06 January 2025More LessLung cancer is a leading cause of mortality worldwide. Immunotherapy has emerged as a potentially effective strategy, as traditional medicines have shown minimal success. This review investigates the current state of immunotherapy for lung cancer treatment, focusing on its mechanisms, clinical applications, strategies, and future directions. This study focuses on the different characteristics of non-small and small-cell lung Read More
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From DNA Editing to RNA Regulation: The Breakthroughs of CRISPR and Mega-CRISPR
Authors: Dinesh Kumar, Debayan Sil, Komal, Balak Das Kurmi and Manish KumarAvailable online: 04 December 2024More Less
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Unraveling Glioblastoma: TME Implication and Gene Therapy Advances
Authors: Rohith Raali and P.K SureshAvailable online: 14 November 2024More LessGlioblastoma is a malignant manifestation of a solid brain tumour with a very dismal prognosis due to an overall median survival of 14 months. The currently administered Standard treatment plan, the STUPP regimen, is not very effective in tackling this neoplasia. A major concern that affects the development of new drug formulations, specifically for Glioma, is the inherent sub-clonal heterogeneity, which includes the dynamic Read More
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Comprehensive Analysis and Experimental Validation of HEPACAM2 as a Potential Prognosis Biomarker and Immunotherapy Target in Colorectal Cancer
Authors: Shouguang Wang, Lijuan Zhang, Dongbing Li and Miaomiao GouAvailable online: 01 November 2024More LessBackground The role of HEPACAM family member 2 (HEPACAM2) is unclear in colorectal cancer (CRC). Objective The objective of this study was to perform an extensive examination of HEPACAM2 and validate it experimentally in CRC. Methods This study investigated the significance of HEPACAM2 in CRC and its potential diagnostic utility utilizing data from the Cancer Genome Atlas (TCGA) database. Additionally, the stud Read More
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Probing the Depths of Molecular Complexity: STAT3 as a Key Architect in Colorectal Cancer Pathogenesis
Available online: 28 October 2024More LessColorectal cancer (CRC) has become a significant threat in recent decades, and its incidence is predicted to continue rising. Despite notable advancements in therapeutic strategies, managing CRC poses complex challenges, primarily due to the lack of clinically feasible therapeutic targets. Among the myriad molecules implicated in CRC, the signal transducer and activator of transcription 3 (STAT3) stands out as a pr Read More
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Gene Augmentation Techniques to Stimulate Wound Healing Process: Progress and Prospects
Authors: Jyotsana Dwivedi, Shubhi Kaushal, D Jeslin, L. Karpagavalli, Rajesh Kumar, Dhruv Dev and Pranay WalAvailable online: 23 October 2024More LessGene therapy has traditionally been used to treat individuals with late-stage cancers or congenital abnormalities. Numerous prospects for therapeutic genetic modifications have emerged with the discovery that gene therapy applications are far more extensive, particularly in skin and exterior wounds. Cutaneous wound healing is a complex, multistep process involving multiple steps and mediators that operate in a net Read More
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New Hope for Intervertebral Disc Degeneration: Bone Marrow Mesenchymal Stem Cells and Exosomes Derived from Bone Marrow Mesenchymal Stem Cell Transplantation
Authors: Xiao-bo Zhang, Xiang-yi Chen, Jin Qi, Hai-yu Zhou, Xiao-bing Zhao, Yi-cun Hu, Rui-hao Zhang, De-chen Yu, Xi-dan Gao, Ke-ping Wang and Lin Ma
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