Results 131 to 140 of about 121,774 (253)
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai +9 more
wiley +1 more source
Rapid production of precisely edited cattle and mice via a single AAV delivered miniature IscB<sup>#</sup>/ωRNA. [PDF]
Song L +14 more
europepmc +1 more source
A Mechanistic Model of rAAV Production in Synthetic Cell Lines
ABSTRACT The recombinant adeno‐associated virus (rAAV) is a widely used vector for gene therapy. Its manufacturing faces significant challenges in producing the large quantities of vectors needed for clinical applications and reducing empty particles.
Prahalad Srinivasan +5 more
wiley +1 more source
The effects of NETs on regeneration of various diabetic tissues, and strategies targeting NETs for diabetes tissue regeneration. In the diabetic environment, NETs undergo complex metabolic and immune reprogramming, leading to dynamic changes in antibacterial and proinflammatory functions, and affecting regeneration of multiple systemic tissues.
Xinyi Jiang +6 more
wiley +1 more source
AAV-Based Bright and Sparse Labeling of Versatile Neurons Adaptable in Cre-Dependent Genetic Backgrounds. [PDF]
Kamijo S, Ikeda K, Miwa H.
europepmc +1 more source
Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks
A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan +8 more
wiley +1 more source
ANCA-Associated Vasculitis with Predominant Peripheral and Central Nervous System Involvement: A Case Report. [PDF]
Zhang B, Jing C, Liu P, Zhang J.
europepmc +1 more source
Traditional dosing strategies often rely on a “one‐size‐fits‐all” paradigm, assuming an “average” patient with typical demographic and pharmacological characteristics. In reality, this often overlooks existing between‐patient variability and can lead to suboptimal drug exposure or toxicity. This issue is especially pronounced in pediatric patients, who
Zachary L. Taylor +12 more
wiley +1 more source
STARR-CRAAVT: A platform to identify cell type-specific regulatory elements for next-generation gene therapy. [PDF]
Becker R +11 more
europepmc +1 more source
AAVrh10 gene therapy ameliorates central and peripheral nervous system disease in canine globoid cell leukodystrophy (Krabbe disease) [PDF]
et al,, Jiang, Xuntain, Ory, Daniel S
core +1 more source

