Results 71 to 80 of about 74,668 (263)

Novel Cytotoxic Vectors Based on Adeno-Associated Virus

open access: yesToxins, 2010
Vectors based on adeno-associated virus (AAV) are promising tools for gene therapy. The production of strongly toxic vectors, for example for cancer-directed gene transfer, is often unfeasible due to uncontrolled expression of toxic genes in vector ...
Johannes Kohlschütter   +2 more
doaj   +1 more source

SR‐A3 Promotes USP18‐Mediated deISGylation of STING to Protect Against Myocardial Ischemia‐Reperfusion Injury

open access: yesAdvanced Science, EarlyView.
MI/RI is a major clinical challenge with high morbidity and mortality. This study demonstrates that SR‐A3 expression is downregulated in MI/RI, and its deletion exacerbates damage via the USP18/ISG15/STING axis, revealing SR‐A3 as a potential therapeutic target.
Yufei Han   +24 more
wiley   +1 more source

A novel polyethyleneimine-coated adeno-associated virus-like particle formulation for efficient siRNA delivery in breast cancer therapy: preparation and in vitro analysis

open access: yesInternational Journal of Nanomedicine, 2012
Wei Shao1, Arghya Paul1, Sana Abbasi1, Parminder S Chahal2, Jimmy A Mena2, Johnny Montes2, Amine Kamen2, Satya Prakash11Biomedical Technology and Cell Therapy Research Laboratory, Department of Biomedical Engineering and Artificial Cells and Organs ...
Shao W   +7 more
doaj  

Compact 9dBEs Enable Efficient and Precise Genome Editing in Mammalian Cells and In Vivo

open access: yesAdvanced Science, EarlyView.
As a compact type II‐D system, the Cas9d‐based platform holds great potential for in vivo applications. Through rational engineering, its derived base editors (9dBEs) enable efficient disease modeling while facilitating single‐vector AAV delivery for in vivo genome editing. These miniature tools offer a robust strategy for basic research and biomedical
Qingquan Xiao   +12 more
wiley   +1 more source

Retinal Penetrating Adeno-Associated Virus

open access: yesInvestigative Ophthalmology & Visual Science
The most common method of delivery of genes to the outer retina uses recombinant adeno-associated virus (AAV) injected into the subretinal space using a surgical procedure. In contrast, most drugs are delivered to the retina using an intravitreal approach in an office setting. The objective of the current study was to develop AAV vectors that can reach
Kumar, Binit   +3 more
openaire   +2 more sources

Gut Microbiota‐Isoallolithocholic Acid Crosstalk Promotes Calcium Oxalate Kidney Stone Formation via PARP1‐Mediated Parthanatos

open access: yesAdvanced Science, EarlyView.
ABSTRACT Bile acids have been implicated in calcium oxalate (CaOx) nephrolithiasis. Here, we employ multi‐omics approaches to identify isoallolithocholic acid (isoalloLCA) as the key bile acid elevated in the feces, serum, kidney, and urine of CaOx rats, confirmed by spatial metabolomics.
Zijian Zhou   +9 more
wiley   +1 more source

Macrophage PABPC4‐SPP1 Axis Orchestrates Immunosuppression in Colorectal Cancer

open access: yesAdvanced Science, EarlyView.
In the CRC microenvironment, macrophage PABPC4 binds to the 3′UTR of SPP1 mRNA to stabilize its expression, thereby sustaining M2‐like immunosuppressive macrophage polarization. Concurrently, this axis suppresses CD8+ T cell effector functions via CD44 signaling, collectively fostering an immunosuppressive niche that drives tumor progression.
Meng Wang   +14 more
wiley   +1 more source

Association between adeno‐associated virus genomic titers and intracellular plasmid levels

open access: yesEngineering Reports
The recombinant adeno‐associated viral (rAAV) vector is one of the most effective viral vectors in gene therapy because of its low immunogenicity, high transduction efficiency, broad tissue specificity, and long‐term transgene expression ability. HEK293T
Kyoko Masumi‐Koizumi   +4 more
doaj   +1 more source

KLF5 Downregulation Links Impaired BNIP3‐Mediated Mitophagy to Inflammatory Valve Remodeling in Calcific Aortic Valve Disease

open access: yesAdvanced Science, EarlyView.
In human CAVD, KLF5 is reduced in VIC‐rich regions and remodeling/stress‐associated VIC states. In VICs, KLF5 sustains BNIP3 promoter activity and BNIP3‐mediated mitophagy, thereby limiting cytosolic mtDNA accumulation. KLF5 loss weakens mitochondrial quality control and enhances mtDNA‐sensitive STING/NF‐κB/NLRP3 inflammatory signaling under osteogenic
Jin‐Hui Bian   +13 more
wiley   +1 more source

Comprehensive mutagenesis maps the effect of all single-codon mutations in the AAV2 rep gene on AAV production

open access: yeseLife
Recombinant adeno-associated viruses (rAAVs) are the predominant gene therapy vector. Several rAAV vectored therapies have achieved regulatory approval, but production of sufficient rAAV quantities remains difficult.
Nina K Jain   +2 more
doaj   +1 more source

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