Long-Term Safety and Efficacy of AAV9 Vectors Expressing Human SMN1 Gene: A Preclinical Study [0.03%]
Vahid Mansouri,Maryam Bemanalizadeh,Naser Ahmadbeigi et al.
Vahid Mansouri et al.
These findings demonstrate that AAV9-hcoSMN therapy achieves sustained and widespread transgene expression with no observable toxicity in a neonatal mouse model, reinforcing the therapeutic potential of AAV9-based gene delivery for SMA.
Exploring Negative Feedback Mechanisms in the PTEN-ACE Axis: Application of Electrosorb Hydrogel-Based Gene Delivery for Intervertebral Disc Regeneration [0.03%]
基于电吸附水凝胶的基因递送在椎间盘再生中PTEN-ACE轴负反馈机制的研究及应用
Youfeng Guo,Feng Wang,Yu Zhou et al.
Youfeng Guo et al.
Intervertebral disc degeneration (IDD), along with associated low back pain, stands as a primary cause of disability. The renin-angiotensin-aldosterone system has been linked to IDD; however, the mechanisms underlying this relationship have...
PET-based tracking of CAR T cells and viral gene transfer using a cell surface reporter that binds to lanthanide complexes [0.03%]
基于PET的CAR-T细胞追踪及利用结合镧系化合物的细胞表面报告基因的病毒介导的基因转移技术
Volker Morath,Katja Fritschle,Linda Warmuth et al.
Volker Morath et al.
This depth-independent whole-body imaging platform offers a powerful tool for monitoring therapeutic cell dynamics and gene delivery in preclinical and potentially clinical settings. © 2025. The Author(s).
Current biological, chemical and physical gene delivery approaches for producing induced pluripotent stem cells (iPSCs) [0.03%]
用于诱导多能干细胞(iPSCs)制备的当前生物、化学和物理基因递送方法
Elahe Izadi,Seyedeh Shima Mohammad,Mazdak Ganjalikhani Hakemi et al.
Elahe Izadi et al.
Due to the importance of iPSCs over the past decade, various studies have focused on finding a safe and efficient gene delivery system to introduce reprogramming factors (RFs) into somatic cells, particularly for clinical applications.
3D Quantification of Viral Transduction Efficiency in Living Human Retinal Organoids [0.03%]
人源视网膜类器官中病毒转导效率的三维定量分析
Teresa S Rogler,Katja A Salbaum,Achim T Brinkop et al.
Teresa S Rogler et al.
The approach combines quantification of gene delivery efficiency in space and time, leveraging human retinal organoids, engineered adeno-associated virus (AAV) vectors, confocal live imaging, and deep learning-based image segmentation....The integration of these tools in an organoid imaging and analysis pipeline allows quantitative testing of future treatments and other gene delivery methods.
Intelligent Gene Delivery System Functionalized Metal Implants for Fracture Repair via Remodeling Mitochondrial Homeostasis [0.03%]
通过调节线粒体稳态功能化金属植入物的智能基因传递系统促进骨折修复
Gang Zheng,Feng Hu,Mengqian Wu et al.
Gang Zheng et al.
In this study, an intelligent gene delivery system is engineered for targeted gene delivery to BMSCs, utilizing diselenide-bridged mesoporous organosilica nanoparticles (SeMONs) modified with a BMSC-affinity peptide (E7)....Compared to conventional mesoporous organosilica nanoparticles, SeMONs exhibit not only superior gene delivery properties but also unique reactive oxygen species (ROS)-responsive degradation and scavenging capabilities....Given that siRNA-Foxf1 (siFoxf1) is known to promote osteogenesis, this gene delivery system carrying siFoxf1 (E7-SeMONs@siFoxf1) is anchored onto metal implants, to create a novel coating designed to promote fracture repair.
Induced Proteinuria Enhances Adeno-Associated Virus Transduction of Renal Tubule Epithelial Cells After Intravenous Administration [0.03%]
诱导蛋白尿增强静脉注射腺相关病毒转导肾小管上皮细胞的能力
Jeffrey D Rubin,Katayoun Ayasoufi,Erin B McGlinch et al.
Jeffrey D Rubin et al.
However, gene delivery to renal tubule epithelial cells mediated by viral vectors via the blood is inefficient due to the permselectivity of the glomerular barrier.
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve [0.03%]
工程化AAV衣壳介导小鼠神经纤维瘤和腓肠神经转导
Edwina Abou Haidar,Shilpa Prabhakar,Pike See Cheah et al.
Edwina Abou Haidar et al.
Gene therapy using adeno-associated virus (AAV) vector mediated gene delivery is a promising strategy to treat these diseases.
Guanosine-Based Supramolecular Particles for Enhanced Drug and Gene Delivery in Cell Culture [0.03%]
鸟苷基超分子颗粒在细胞培养中的增强药物和基因传递作用
Luis M Negrón,Edwin Vázquez-Rosa,Luxene Belfleur et al.
Luis M Negrón et al.
Supramolecular hacky sacks (SHS) are a distinct class of self-assembled colloidal particles derived from guanosine (G) derivatives, engineered to support a wide range of cellular and therapeutic functions. In this study, we examine how vari...
Novel PEI-aldehyde conjugates for gene delivery: Promoting chondrogenic differentiation in human mesenchymal stem cells [0.03%]
用于基因传递的新型聚乙烯亚胺醛化衍生物：促进人源间充质干细胞软骨分化效应及机制研究
Diego Miranda-Balbuena,Alba Ramil-Bouzas,Naiara Doldán-Mata et al.
Diego Miranda-Balbuena et al.
Still, finding appropriate vectors to achieve a safe and efficient gene delivery remains a challenge....These findings suggest that PEIT2T3A is a promising non-viral vector for targeted gene delivery and hMSC-based regenerative medicine applications.
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