GH-resistant (Laron) mice: gene therapy with a liver-specific GH receptor causes unbalanced upregulation of female-biased and growth-related genes. [PDF]
Tay JK +9 more
europepmc +1 more source
Objective To assess the 2022 American College of Rheumatology (ACR)/EULAR classification criteria for antineutrophil cytoplasmic antibody–associated vasculitis (AAV) in children with chronic small‐to‐medium vessel vasculitis. Methods A cohort of 574 patients, identified by physician's diagnosis (MD‐diagnosis) in A Registry of Childhood Vasculitis, was ...
David A. Cabral +41 more
wiley +1 more source
Precision medicine in paediatrics: Progress and priorities
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain +3 more
wiley +1 more source
Large-scale proteome profiling identifies circulating biomarkers for disease activity and organ involvement in ANCA-associated vasculitides. [PDF]
Hellbacher E +13 more
europepmc +1 more source
What Makes an “Ideal” Cell Line for Recombinant Adeno‐Associated Virus Production?
Several host cell types have been used to produce rAAVs to date. Cell line‐specific traits that are beneficial in the context of rAAV manufacturing are reviewed here, with the goal of developing a consensus on the ‘ideal’ characteristics that an rAAV production host should possess (created with Biorender.com).
James Conheady +6 more
wiley +1 more source
A High-Throughput Automation Platform for Accelerated AAV Stability Optimization. [PDF]
Li S, Wang X, Zhi L, Shameem M, Liu D.
europepmc +1 more source
Fouling‐Free Inertial Microfluidic Cell Clarification for Lentivirus Manufacturing
ABSTRACT Lentiviral vectors (LVVs) are widely used as gene‐delivery vehicles in cell and gene therapy, enabling stable integration of transgenes into target cells. However, efficient clarification of LVV harvest remains a major bottleneck in large‐scale manufacturing, as conventional depth filtration often results in significant product loss.
Dohyun Park +5 more
wiley +1 more source
Multi-omics profiling reveals epidermal growth factor as a potential biomarker and therapeutic target in lupus nephritis and ANCA-associated vasculitis with rapidly progressive glomerulonephritis. [PDF]
Xu M +8 more
europepmc +1 more source

