Base Editing: unlocking safer, precise, and complex genome engineering for therapeutics and discovery
Il 26/06/2025 ore 14.30 - 16.00
Via Pietro Castellino n. 111 Napoli c/o Area di Ricerca Napoli 1- Conference Room
CRISPR-based technologies have transformed genome engineering, yet their reliance on double-strand DNA breaks (DSBs) poses challenges, particularly for applications requiring precision, safety, and multiplexing. Base editors have emerged as next-generation tools, enabling efficient and precise single nucleotide changes without introducing DSBs. In this seminar, I will introduce the principles of CRISPR-based genome engineering with a particular focus on base editing and its expanding role in therapeutic and research applications.
I will highlight Revvity’s Pin-point™ platform, a modular base editing system that leverages RNA aptamer-mediated assembly to deliver flexible, efficient, and specific base editing. This unique architecture enables sophisticated editing strategies—such as multiplexed gene knockouts and simultaneous knockout and transgene integration—while minimizing undesired effects on genome integrity.
Therapeutic applications of the Pin-point™ platform include streamlined generation of next-generation allogeneic CAR-T cells and hypoimmunogenic iPSCs, as well as efficient editing in sensitive stem cell populations like HSPCs and precise correction of pathogenic SNVs. In addition to its therapeutic potential, I will describe how base editing can be applied to functional genomics, enabling high-resolution pooled mutagenesis screens to map genotype-phenotype relationships, identify drug-variant interactions, and accelerate target discovery.
Together, these developments position base editing—and the Pin-point™ platform in particular—as a versatile platform poised to advance both precision medicine and fundamental genetic research.
Disclaimer:
Pin-point™ Technology Platform: The Pin-point™ base editing platform technology is available for clinical or diagnostic study and commercialization under a commercial license from Revvity
Organizzato da:
Istituto di biochimica e biologia cellulare (Cnr-Ibbc)
Referente organizzativo:
Pina Marotta
CNR - Istituto di biochimica e biologia cellulare
via Pietro Castellino n. 111 Napoli
pina.marotta@cnr.it
Modalità di accesso: ingresso libero
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