gene-therapy-targeting
AAV-PR-CBA-Cre mediates vasculature-tropic transduction and gene modification in transgenic Ai9 mice (CAG-floxed-STOP-tdTomato). (A) AAV-PR peptide and insertion site. The peptide PRPPSTH is inserted after amino acid 588 of AAV9 VP1. A Cre-recombinase expression cassette is packaged inside AAV-PR and this is administered systemically to Ai9 mice, which contain a floxed-stop-tdTomato cassette in all cells. If AAV-PR mediates Cre expression, the loxP-stop is removed from the genome allowing tdTomato expression. (B) Half brain hemisphere of Ai9 mouse injected i.v. with AAV-PR-CBA-Cre showing bright intrinsic fluorescence (white signal). Letters (C–F) indicate areas of zoomed images in (B). The cells that were transduced had morphology consistent with the vasculature (pink arrowheads), and neurons (yellow and blue arrowheads). Scale bar in (B) = 500 μm. (C–F) = 10 μm. AAV, adeno-associated virus; CBA, hybrid CMV early/chicken β-actin promoter; i.v., intravenous. Credit: Human Gene Therapy (2023). DOI: 10.1089/hum.2022.211

Researchers have developed an engineered adeno-associated virus (AAV) vector that yields high transduction of brain vascular pericytes and smooth muscle cells. The study describing the characterization of this novel AAV capsid is published in the journal Human Gene Therapy.

In the current study, Servio Ramirez, from Temple University School of Medicine, Patricia Musolino, from Massachusetts General Hospital, and Casey Maguire, from Harvard Medical School, and coauthors, characterize AAV-PR, the capsid that demonstrated high transduction of the brain vasculature.

AAV-PR offers the possibility of genetically modulating brain pericytes and smooth muscle cells in the context of neurodegeneration and other neurological diseases, according to the investigators. Many common neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, and amyotrophic lateral sclerosis, involve the brain vasculature.

“Because so many neurologic conditions stem from vascular dysfunction, the ability to deliver genes to the cells comprising these vessels could be truly paradigm shifting,” says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Chan Medical School.

Source: https://medicalxpress.com/news/2023-08-gene-therapy-brain-vasculature.html
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PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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