Results 111 to 120 of about 9,649,206 (293)

Adeno-Associated Virus-Based Vectors in Gene Therapy

Journal of Biomedical Science, 2000
Adeno-associated virus (AAV) vectors were shown capable of high efficiency transduction of both dividing and nondividing cells and tissues. AAV-mediated transduction leads to stable, long-term transgene expression in the absence of apparent immune response.
openaire   +2 more sources

Adeno-associated virus vectors: versatile tools for in vivo gene transfer.

, 2008
Over the last few years, viral vectors based on the adeno-associated virus have gained increasing popularity due to several favorable characteristics, including the high efficiency of transduction of postmitotic tissues in vivo and the long-term ...
Mauro Giacca, L. Zentilin, Lorena Zentilin, GIACCA, MAURO   +3 more
core   +1 more source

SIRT7‐Mediated H2BK120 Succinylation Drives Aberrant Mitophagy in Sepsis‐Associated Cognitive Dysfunction

Advanced Science, EarlyView.
Sepsis triggers hippocampal SIRT7 loss, promoting histone H2B succinylation and activation of the PD‐1/PD‐L1–PINK1 axis. This metabolic‐epigenetic cascade drives aberrant mitophagy and neuronal injury, ultimately causing cognitive deficits. Targeting SIRT7‐dependent succinylation offers a potential strategy to protect brain function during sepsis ...
Na Meng, Jiateng Zhou, Xiaoyu Guo, Ting Hong, Xuelian Li, Xingyu Wei, Chanhua Zhang, Xixue Zhang, Songbin Liu, Yulin Zhang, Liangfang Yao, Lina Huang, Weidong Gu   +12 more
wiley   +1 more source

Gender difference in pre-clinical liver-directed gene therapy with lentiviral vectors

Experimental Biology and Medicine
Viral vector-based therapies are effective therapeutics for the correction of several disorders, both in mouse models and in humans. Several pre-clinical studies have demonstrated differences in transduction efficiencies and therapeutic effect between ...
Efrain Guzman, Cheen Khoo, Deirdre O’Connor, Gayathri Devarajan, Sharifah Iqball, Bernard Souberbielle, Kyriacos Mitrophanous, Yatish Lad   +7 more
doaj   +1 more source

Engineered Transformer Base Editor with Enhanced Editing Efficiency

Advanced Science, EarlyView.
A highly efficient transformer base editor system achieves robust genomic editing in a humanized mouse model. This work establishes a versatile and translatable platform, opening new avenues for precision gene therapy. ABSTRACT Canonical cytosine base editors (CBEs) achieve precise C‐to‐T conversions without inducing DNA double‐strand breaks (DSBs ...
Bowen Chen, Letong Liang, Rui Xu, Tianyi Wang, Hongyan Chen, Daru Lu   +5 more
wiley   +1 more source

FTSJ3‐Mediated 2′‐O‐Methylation of HSPA5 mRNA Stabilizes BOP1 via SUMOylation to Drive DRP1‐Dependent Mitochondrial Fission and Fatty Acid Oxidation in Lung Squamous Cell Carcinoma

Advanced Science, EarlyView.
The RNA methyltransferase FTSJ3 drives lung squamous cell carcinoma progression by catalyzing 2′‐O‐methylation of HSPA5 mRNA, which stabilizes BOP1 through SUMOylation and activates DRP1/SREBP1‐mediated mitochondrial fission and fatty acid oxidation. Targeting this axis with liposomal siRNA potently suppresses tumor growth and metastasis ABSTRACT Lung ...
Qin Hu, Jiaheng Lin, Yifei Zhu, Ying Liu, Guoren Zhou   +4 more
wiley   +1 more source

Probing aspects of extracellular vesicle associated AAV allows increased vector yield and insight into its transduction and immune-evasive properties

Molecular Therapy: Methods & Clinical Development
Extracellular vesicle-associated adeno-associated virus vectors (EV-AAVs) are generated during production in 293 cells. EV-AAV provides desirable gene delivery traits such as greater resistance to antibody neutralization and increased transduction of ...
Ming Cheng, Demitri de la Cruz, Adam V. Crain, Paula Espinoza, Carrie Ng, Zachary C. Elmore, Aravind Asokan, Casey A. Maguire   +7 more
doaj   +1 more source

The GPX3‐VCAM1 Axis Gates Pro‐Fibrotic Tubule Cell Fate in Hyperuricemic Nephropathy

Advanced Science, EarlyView.
Hyperuricemia induces NLRP3 inflammasome signaling activation and downregulation of GPX3 in proximal tubular epithelial cells, leading to oxidative stress–driven VCAM1 upregulation, emergence of pro‐fibrotic proximal tubule cells, macrophage recruitment, and renal fibrosis.
Yunfei Qi, Qiang Zhao, Yue Lve, Yaming Yuan, Tingting Zhou, Yue Yuan, Dexi Wu, Yifan Tang, Yan Liu, Zhigang Li, Chunping Jiang, Qiurong Ding   +11 more
wiley   +1 more source

Stable Producer Cell Lines for Adeno-Associated Virus (AAV) Assembly

, 2010
INTRODUCTIONStable producer cell lines containing both the rep and cap genes and recombinant adeno-associated virus (rAAV) vectors can be infected with a helper virus to provide reliable and efficient production of rAAV stocks.
Anna Salvetti, Gilliane Chadeuf
core   +1 more source

CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome

Advanced Science, EarlyView.
By modeling Lowe syndrome using patient‐derived iPSCs, this study establishes a human disease model that faithfully recapitulates OCRL deficiency‐associated ciliary and cytoskeletal defects. The model enables evaluation of both mutation‐agnostic DNA augmentation and CRISPR‐mediated mutation correction strategies, both of which restore OCRL function and
Siyu Chen, Zhiquan Liu, Wenmin Wang, Qing Wang, Tia J. Kowal, Fan Zhang, Grzegorz Walkiewicz, Faruk Hossen, Herbert M. Lachman, Jinqiong Zhou, Yiting Wang, Yang Sun   +11 more
wiley   +1 more source