Retroviral and Lentiviral Vectors for the Induction of Immunological Tolerance by Inès Dufait, Therese Liechtenstein, Alessio Lanna, Christopher Bricogne, Roberta Laranga, Antonella Padella, Karine Breckpot, David Escors

“…They have been used for some time for gene therapy and the development of gene vaccines. Recently retroviral and lentiviral vectors have been used to generate tolerogenic dendritic cells, key professional antigen presenting cells that regulate immune responses. …”
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Local Gene Transfer of OPG Prevents Joint Damage and Disease Progression in Collagen-Induced Arthritis by Qingguo Zhang, Weiming Gong, Bin Ning, Lin Nie, Paul H. Wooley, Shang-You Yang

“…Overall, gene transfer of OPG effectively inhibited the arthritis-associated periarticular bone erosion and preserved the architecture of arthritic joints, and the study provides evidence that the cartilage protection of the OPG gene therapy may be associated with the down-regulation of MMP3 expression.…”
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Targeting ion homeostasis in metabolic diseases: Molecular mechanisms and targeted therapies by Yanjiao Zhang, Kaile Ma, Xinyi Fang, Yuxin Zhang, Runyu Miao, Huifang Guan, Jiaxing Tian

“…Lastly, the article discusses the development direction of future therapeutic strategies, including the possibility of gene therapy targeting specific ion channels and personalized therapy using novel biomarkers. …”
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Adenovirus Vectors Target Several Cell Subtypes of Mammalian Inner Ear In Vivo by Yilai Shu, Yong Tao, Wenyan Li, Jun Shen, Zhengmin Wang, Zheng-Yi Chen

“…This study shows that adenovirus vectors are capable of efficiently and specifically transducing different cell types in the mammalian inner ear and provides useful tools to study inner ear gene function and to evaluate gene therapy to treat hearing loss and vestibular dysfunction.…”
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Construction and Antiapoptosis Activities of Recombinant Adenoviral Expression Vector Carrying EBV Latent Membrane Protein 2A by Xishuang Liu, Yu Gao, Bing Luo, Youan Zhao

“…This study establishes a foundation for further study on EBVaGC and its gene therapy.…”
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Cranial Cruciate Ligament Desmotomies in Sheep Resulting in Peroneus Tertius Injury by Peter J. Welsh, Crystal G. Collier, Holly M. Clement, Michael N. Vakula, Jeffrey B. Mason

“…Eight sheep underwent CCLD for use in a gene therapy study. We report this case in which six of the eight sheep were clinically diagnosed by pathognomonic signs and later confirmed by postmortem dissection, with injury of the peroneus tertius (PT) muscle. …”
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Optogenetic regulation of endogenous gene transcription in mammals by E. S. Omelina, A. V. Pindyurin

“…However, finding ways to control the activity of endogenous genes is absolutely necessary for further progress in safe and effective gene therapies and regenerative medicine. In addition, such systems are of particular interest for genetics, molecular and cell biology. …”
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Challenges and Innovations in Alveolar Bone Regeneration: A Narrative Review on Materials, Techniques, Clinical Outcomes, and Future Directions by Diana Marian, Giuseppe Toro, Giovanbattista D’Amico, Maria Consiglia Trotta, Michele D’Amico, Alexandru Petre, Ioana Lile, Anca Hermenean, Anca Fratila

“…It examines traditional techniques like guided bone regeneration and bone grafting, alongside newer methods such as stem cell therapy, gene therapy, and 3D bioprinting. Each approach is considered for its strengths in supporting bone growth and integration, especially in cases where complex bone defects make regeneration difficult. …”
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Candidate antirheumatic genotherapeutic plasmid constructions have low immunogenicity by T. S. Nepomnyashchikh, T. V. Tregubchak, S. N. Yakubitskiy, O. S. Taranov, R. A. Maksyutov, S. N. Shchelkunov

“…Viral genes coding for immunomodulatory factors could be used to create new gene therapy products to treat RA and other human disease. …”
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CRISPR-Cas9 in basic and translational aspects of cancer therapy by Maryam Samareh Salavatipour, Zahra Poursalehi, Negin Hosseini Rouzbahani, Sohaib Mohammadyar, Mohammad Vasei

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Exosomal miRNAs involvement in pathogenesis, diagnosis, and treatment of rheumatoid arthritis by Mahvash Sadeghi, Jalil Tavakol Afshari, Afsane Fadaee, Mohammadreza Dashti, Fatemeh Kheradmand, Sajad Dehnavi, Mojgan Mohammadi

“…It highlighted present situation and future perspective of using exosomal miRNAs as biomarkers and a specific gene therapy approach for RA treatment.…”
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4-Hydroxycinnamic acid attenuates neuronal cell death by inducing expression of plasma membrane redox enzymes and improving mitochondrial functions by Sujin Park, Yoon A Kim, Jaewang Lee, Hyunsoo Seo, Sang-Jip Nam, Dong-Gyu Jo, Dong-Hoon Hyun

“…Many approaches to neurodegenerative diseases that focus on amyloid-β clearance and gene therapy have not been successful. Some therapeutic applications focus on enhancing neuronal cell survival during the pathogenesis of neurodegenerative diseases, including mitochondrial dysfunction. …”
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Hepatoma-Targeted Radionuclide Immune Albumin Nanospheres: 131I-antiAFPMcAb-GCV-BSA-NPs by Mei Lin, Junxing Huang, Dongsheng Zhang, Xingmao Jiang, Jia Zhang, Hong Yu, Yanhong Xiao, Yujuan Shi, Ting Guo

“…Due to good drug-loading, high encapsulation ratio, and highly selective affinity for AFP-positive tumors, the 131I-antiAFPMcAb-GCV-BSA-NPs are promising for further effective radiation-gene therapy of hepatoma.…”
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GOLPH3 inhibits glioma cell apoptosis through the JNK signaling pathway by Shao Xie, Shao Xie, Jiahai Ding, Zhaohao Wang, Hengliang Shi, Zheng-Quan Yu

“…Understanding the molecular mechanisms underlying glioma initiation and progression and identifying potential therapeutic targets for gene therapy are crucial for improving patient outcomes. …”
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CRISPR/Cas9, a universal tool for genomic engineering by A. V. Smirnov, A. M. Yunusova, V. A. Lukyanchikova, N. R. Battulin

“…Since Cas9-related hype is mostly driven by its remarkable potential for gene therapy and genome engineering, the latest CRISPR/Cas9 applications in these areas are also covered in our review. …”
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Mesenchymal Stromal Cell Therapy in Ischemia/Reperfusion Injury by Pascal Rowart, Pauline Erpicum, Olivier Detry, Laurent Weekers, Céline Grégoire, Chantal Lechanteur, Alexandra Briquet, Yves Beguin, Jean-Marie Krzesinski, François Jouret

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Bisphosphonate Treatment in a Patient Affected by MPS IVA with Osteoporotic Phenotype by Albina Tummolo, Orazio Gabrielli, Alberto Gaeta, Maristella Masciopinto, Lucia Zampini, Luigi Michele Pavone, Paola Di Natale, Francesco Papadia

“…No treatment with allogenic bone marrow transplantation or gene therapy is currently available for Morquio A syndrome, and enzyme replacement therapy is under evaluation. …”
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Corrigendum to “Depletion of ß1,6-N-acetylglucosaminyltransferase reduces E-selectin binding capacity and migratory potential of human gastrointestinal adenocarcinoma cells” [Neopl... by Lisa Staffeldt, Hanna Maar, Julia Beimdiek, Samuel Chambers, Kristoffer Riecken, Mark von Itzstein, Falk FR Buettner, Arun Everest-Dass, Tobias Lange

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Impact of hydroxyurea on clinical and biological parameters of sickle cell anemia in children in Abidjan by MIREILLE YAYO- AYE, Adia Eusèbe Adjambri, Boidy Kouakou, Rebecca N'guessan-Blao, Louis Missa Adjé, Taïratou Kamagaté, Vincent Yapo, Duni Sawadogo

“…Curative treatments such as bone marrow transplantation or gene therapy exist, but are not currently performed in Côte d'Ivoire. …”
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Identification of Potential Clusters of Signs and Symptoms to Prioritize Patients’ Eligibility for AADCd Screening by 3-OMD Testing: An Italian Delphi Consensus by Carlotta Spagnoli, Roberta Battini, Filippo Manti, Duccio Maria Cordelli, Andrea Pession, Melissa Bellini, Andrea Bordugo, Gaetano Cantalupo, Antonella Riva, Pasquale Striano, Marco Spada, Francesco Porta, Carlo Fusco

“…AADCd is an ultrarare, underdiagnosed neurometabolic disorder for which a screening test (3-OMD dosing on dried blood spot (DBS)) and targeted gene therapy (authorized in the EU and the UK) are available. …”
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