Filtering through AAV Capsid Libraries for Effective Kidney Gene Transfer

Kidney International, Journal Year: 2025, Volume and Issue: unknown

Published: Jan. 1, 2025

Language: Английский

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Gene Editing: An Effective Tool for the Future Treatment of Kidney Disease

Journal of Inflammation Research, Journal Year: 2025, Volume and Issue: Volume 18, P. 4001 - 4018

Published: March 1, 2025

Gene editing technology involves modifying target genes to alter genetic traits and generate new phenotypes. Beginning with zinc-finger nucleases (ZFN) transcription activator-like effector (TALEN), the field has evolved through advent of clustered regularly interspaced short palindromic repeats CRISPR-associated protein (CRISPR-Cas) systems, more recently base editors (BE) prime (PE). These innovations have provided deep insights into molecular mechanisms complex biological processes paved way for novel therapeutic strategies a range diseases. is now being applied in treatment both acquired kidney diseases, as well transplantation correction mutations. This review explores current applications mainstream gene technologies biology, particular emphasis on their roles disease research of. It also addresses limitations challenges associated these technologies, while offering perspectives future potential this field.

Language: Английский

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Enhancing gene transfer to renal tubules and podocytes by context-dependent selection of AAV capsids

Nature Communications, Journal Year: 2024, Volume and Issue: 15(1)

Published: Dec. 30, 2024

AAV vectors show promise for gene therapy; however, kidney transfer remains challenging. Here we conduct a barcode-seq-based comparison of 47 capsids administered through different routes in mice, followed by individual validation. We find that local delivery AAV-KP1, but not AAV9, via the renal vein or pelvis effectively transduces proximal tubules with minimal off-target liver transduction, while systemic enhances tubule and podocyte transduction chronic disease. demonstrate these contrasting observations are partly due to differences their pharmacokinetics. Importantly, injection overcomes pre-existing immunity, leading robust exclusive non-human primates (NHPs). In addition, highlight drastic profiles between mice NHPs. Thus, this study provides mechanistic insights underscores importance context-dependent selection overcome challenges kidney.

Language: Английский

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Application of the Insect Cell-Baculovirus Expression Vector System in Adeno-Associated Viral Production

Applied Sciences, Journal Year: 2024, Volume and Issue: 14(23), P. 10948 - 10948

Published: Nov. 25, 2024

Insect Cell-Baculovirus Expression Vector System (IC-BEVS) is an efficient protein expression platform, which famous for its high-level of complex in insect cells. The system based on baculoviruses such as Autographa californica multiple nucleopolyhedrovirus (AcMNPV), and the efficiency target proteins has been significantly improved by optimizing viral vectors cell lines. In recent years, IC-BEVS have shown great potential Adeno-Associated Virus (AAV) production, particularly excelling AAV structural recombinant production. not only improves yield purity AAV, but also shortens production cycle, providing reliable tool gene therapy. However, some challenges, including modification differences, limitations levels, costs. This paper reviews development baculovirus system, application prospects therapy, aiming to provide a systematic reference outlook research related fields.

Language: Английский

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New AAV vector targets glomerular endothelial cells

Nature Reviews Nephrology, Journal Year: 2024, Volume and Issue: unknown

Published: Sept. 27, 2024

Language: Английский

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