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BEGIN:VEVENT
CATEGORIES:Miscellaneous
LOCATION:
STATUS:TENTATIVE
DTSTART:20251119T150000Z
DTEND:20251119T150000Z
TITLE:Transposase- Based Genome Editing: Advantages and Delivery to Treat Retinal Genetic Disease
SUMMARY:Transposase- Based Genome Editing: Advantages and Delivery to Treat Retinal Genetic Disease
DESCRIPTION:Genome editing therapies like CRISPR technologies can mainly correct point mutations. An editing system that could restore function of an entire gene (with multiple mutations) would be beneficial. We present a mammalian transposase that delivers large DNA pieces without inducing double-strand breaks, enhancing safety. Key challenges with this technology are the delivery of twopayloads, minimizing off- target biodistribution, while ensuring a high cellular uptake/expression.  Lipid nanoparticles (LNPs) were engineered to co-deliver DNA and transposase mRNA. Optimized LNPs exhibited improved tolerability, efficient uptake, and considerable ABCA4 expression in Abca4⁻/⁻ mice photoreceptors, suggesting therapeutic efficacy in retinal diseases.
Chair: - Mathew Cherian PhD, Consultant, Member IPS ExCo, USA
Co-moderator: - Qurrotul Aini Shodiquna, Erasmus Mundus Student in Sustainable Drug Discovery, Indonesia
Panellists: - Sandeep Nema PhD, President, BioPharm CMC Advisors, USA - Subhro Guhathakurta PhD, Consultant, USA

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