Divergence in photoreceptor cell death and neuroinflammation in transvitreal and transscleral subretinal delivery in mice by Daniel E. Maidana, Sara Pastor Puente, Catherine Wang, Shivam Chandra, Lucia Gonzalez-Buendia, Eleftherios Paschalis Ilios, Andrius Kazlauskas, Demetrios G. Vavvas

“…Abstract Subretinal injections provide direct access to photoreceptors and RPE, which is crucial for the delivery of gene therapy and neuroprotective approaches. To access the subretinal space, transvitreal (TV) and transscleral (TS) subretinal injections have been widely used in humans and animal models. …”
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High-Fat Diet Augments VPAC1 Receptor-Mediated PACAP Action on the Liver, Inducing LAR Expression and Insulin Resistance by Masanori Nakata, Boyang Zhang, Yifei Yang, Takashi Okada, Norihito Shintani, Hitoshi Hashimoto, Toshihiko Yada

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Stem Cells: A Historical Review about Biological, Religious, and Ethical Issues by Ioannis Alexandros Charitos, Andrea Ballini, Stefania Cantore, Mariarosaria Boccellino, Marina Di Domenico, Elisa Borsani, Riccardo Nocini, Michele Di Cosola, Luigi Santacroce, Lucrezia Bottalico

“…The ethical-scientific debate on this type of cells has taken on great interest both for their application in regenerative medicine and for the potential possibilities in the field of cell and gene therapy. Different points of view often have the expression of a perception that depends on scientific goals or opportunities or on religious traditions and beliefs. …”
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Non-Drug and Non-Invasive Therapeutic Options in Alzheimer’s Disease by Alina Simona Șovrea, Adina Bianca Boșca, Eleonora Dronca, Anne-Marie Constantin, Andreea Crintea, Rada Suflețel, Roxana Adelina Ștefan, Paul Andrei Ștefan, Mădălin Mihai Onofrei, Christoph Tschall, Carmen-Bianca Crivii

“…Adipose-derived stem Cells (ADSCs), gene therapy, and many other alternative methods (diet, sleep pattern, physical exercise, nanoparticle delivery) are also new potential treatments since multimodal approaches represent the modern trend in this disorder research therapies.…”
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Recent advances in nanotechnology for Parkinson’s disease: diagnosis, treatment, and future perspectives by Virendra Kumar Yadav, Seshathiri Dhanasekaran, Nisha Choudhary, Deepak Nathiya, Vishal Thakur, Rachna Gupta, Sheersha Pramanik, Pankaj Kumar, Nishant Gupta, Ashish Patel

“…It further discusses the application of gene therapy in conjunction with nanomedicine for targeted therapeutic interventions.…”
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Pathogenetic therapeutic approaches for endocrine diseases based on antisense oligonucleotides and RNA-interference by Olga Golounina, Ildar Minniakhmetov, Ramil Salakhov, Rita Khusainova, Ekaterina Zakharova, Igor Bychkov, Natalia Mokrysheva

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Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington’s disease by Kaili Ou, Qingqing Jia, Dandan Li, Shihua Li, Xiao-Jiang Li, Peng Yin

“…Currently, available treatments for various neurodegenerative diseases can alleviate symptoms but do not provide a definitive cure. Gene therapy, which aims to modify or express specific proteins for neuroprotection or correction, is considered a powerful tool in managing neurodegenerative conditions. …”
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Harnessing nanotechnology for cancer treatment by Jiajun Zhu, Jiajun Zhu, HaeJu Lee, HaeJu Lee, Ruotong Huang, Ruotong Huang, Jianming Zhou, Jingjun Zhang, Xiaoyi Yang, Wenhan Zhou, Wangqing Jiang, Shuying Chen

“…Furthermore, nanotechnology-based therapeutic strategies, including photothermal therapy, gene therapy and immunotherapy are offering novel ways to combat cancer by selectively targeting tumor cells and enhancing the immune response. …”
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In silico methods for immunogenicity risk assessment and human homology screening for therapeutic antibodies by Aimee E. Mattei, Andres H. Gutierrez, Soorya Seshadri, Jacob Tivin, Matt Ardito, Amy S. Rosenberg, William D. Martin, Anne S. De Groot

“…Although this perspective discusses the in-silico immunogenicity risk assessment for monoclonal antibodies, bi-specifics, multi-specifics, and antibody-drug conjugates, the analysis of additional therapeutic modalities such as enzyme replacement proteins, blood factor proteins, CAR-T, gene therapy products, and peptide drugs is also made available on the ISPRI platform.…”
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DNM3OS Enhances the Apoptosis and Senescence of Spermatogonia Associated with Nonobstructive Azoospermia by Providing miR-214-5p and Decreasing E2F2 Expression by Rui Hua, Qingjun Chu, Feiyan Guo, Qinjie Chen, Maocai Li, Xuan Zhou, Yongtong Zhu

“…DNM3OS was found to regulate the apoptosis and senescence of spermatogonia by providing miR-214-5p and decreasing E2F2 expression, suggesting it as a novel target for gene therapy of male infertility.…”
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Interfacing exogenous stents with human coronary artery by self-assembled coating: designs, functionalities and applications by Feng Zhao, Feng Liu, Chenglong Gao, Guoqing Wang, Yinfeng Zhang, Fei Yu, Jiawei Tian, Kai Tan, Runhao Zhang, Kang Liang, Zhexun Lian, Junjie Guo, Biao Kong, Junbo Ge, Hui Xin

“…Additionally, we examine how self-assembled coatings have been used to release active molecules for anti-coagulation and deliver gene therapy. Moreover, we discuss the potential of self-assembled coatings for future development, including intelligent targeted drug delivery, bionic stent coatings, and 3D printed stent coatings. …”
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An adenoviral vector encoding an inflammation-inducible antagonist, HMGB1 Box A, as a novel therapeutic approach to inflammatory diseases by Kari Ann Shirey, John Joseph, Lynda Coughlan, Haye Nijhuis, Alan W. Varley, Jorge C. G. Blanco, Stefanie N. Vogel

“…We hypothesized that gene therapy platforms could be useful in these cases if the production of an anti-inflammatory protein reflects the intensity and duration of the inflammatory condition. …”
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Mitochondrial-targeted therapies in traumatic brain injury: From bench to bedside by Sidra Tabassum, Silin Wu, Chang-Hun Lee, Bosco Seong Kyu Yang, Aaron M. Gusdon, Huimahn A. Choi, Xuefang S. Ren

“…We also evaluate various preclinical studies exploring pharmacological agents, gene therapy, and novel drug delivery systems designed to protect and restore mitochondrial function. …”
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Therapeutic Potential of HGF-Expressing Human Umbilical Cord Mesenchymal Stem Cells in Mice with Acute Liver Failure by Yunxia Tang, Qiongshu Li, Fanwei Meng, Xingyu Huang, Chan Li, Xin Zhou, Xiaoping Zeng, Yixin He, Jia Liu, Xiang Hu, Ji-Fan Hu, Tao Li

“…Human umbilical cord-derived mesenchymal stem cells (UCMSCs) are particularly attractive cells for cellular and gene therapy in acute liver failure (ALF). However, the efficacy of this cell therapy in animal studies needs to be significantly improved before it can be translated into clinics. …”
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TCR Gene Transfer: MAGE-C2/HLA-A2 and MAGE-A3/HLA-DP4 Epitopes as Melanoma-Specific Immune Targets by Trudy Straetemans, Mandy van Brakel, Sabine van Steenbergen, Marieke Broertjes, Joost Drexhage, Joost Hegmans, Bart N. Lambrecht, Cor Lamers, Pierre van Der Bruggen, Pierre G. Coulie, Reno Debets

“…Further development of TCR gene therapy requires the implementation of T-cell target epitopes that prevent “on-target” reactivity towards healthy tissues and at the same time direct a clinically effective response towards tumor tissues. …”
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The proneural transcription factor Atoh1 promotes odontogenic differentiation in human dental pulp stem cells (DPSCs) by Camila Sabatini, Huey-Jiun Lin, Galib Ovik, Richard Hall, Techung Lee

“…Abstract Background Bioengineering of human teeth for replacement is an appealing regenerative approach in the era of gene therapy. Developmentally regulated transcription factors hold promise in the quest because these transcriptional regulators constitute the gene regulatory networks driving cell fate determination. …”
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A Novel Approach for In Vitro Testing and Hazard Evaluation of Nanoformulated RyR2-Targeting siRNA Drugs Using Human PBMCs by Valeria Bettinsoli, Gloria Melzi, Angelica Crea, Lorenzo Degli Esposti, Michele Iafisco, Daniele Catalucci, Paolo Ciana, Emanuela Corsini

“…As proof of concept, a small interfering RNA (siRNA) targeting Ryanodine Receptor mRNA (RyR2) identified by the Italian National Center for Gene Therapy and Drugs based on RNA Technology as a potential therapeutic target for dominant catecholaminergic polymorphic ventricular tachycardia, was selected. …”
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Ex Vivo Expansion of Murine MSC Impairs Transcription Factor-Induced Differentiation into Pancreatic β-Cells by Dario Gerace, Rosetta Martiniello-Wilks, Rosaline Habib, Binhai Ren, Najah Therese Nassif, Bronwyn Anne O’Brien, Ann Margaret Simpson

“…The data suggest that ex vivo expanded MSCs lose their multipotent differentiation potential and may be more useful as gene therapy targets prior to expansion.…”
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Synergistic strategies for glioblastoma treatment: CRISPR-based multigene editing combined with immune checkpoint blockade by Xiaolin Liu, Xiao Liu, Xiaonan Luo, Maorong Zhu, Nannan Liu, Juan Li, Qi Zhang, Cheng Zou, Yuxin Wu, Zhengcong Cao, Shuangxin Ma, Weizhong Wang, Guangzhao Yang, Jintao Gu, Wei Liu, Meng Li, Anan Yin, Yalong He, Wei Lin

“…Furthermore, the EVs contained Cas9 protein and sgRNA for precise and minimally invasive gene therapy, which addressing the key barriers associated with in vivo CRISPR‒Cas9 gene editing treatment. …”
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Virus-inspired biogenic delivery system for advancing cancer therapy by Di Sun, Hao Liang, Qianwen Mu, Chengchao Chu, Gang Liu, Chao Liu

“…Virus-inspired particles have been utilized in various applications, including vaccination, gene therapy, drug therapy, and diagnostics. Biogenic delivery systems imitating the natural structure of viruses are regarded as innovative nanoplatforms used to deliver drug compounds to related sites and target cells in organisms. …”
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