Transposase- Based Genome Editing: Advantages and Delivery to Treat Retinal Genetic Disease

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