RESEARCH

CRISPR nanoparticles for in-vivo gene therapy

a The revolutionary CRISPR–Cas genome editing technology has enormous potential for effective therapy of many incurable diseases. However, transforming CRISPR into a clinically relevant therapy, which provides safe, targeted, and highly efficient gene editing, is far beyond the capabilities of currently available gene therapy delivery technologies. We are developing a unique nanoplatform that provides a multi-modal toolbox for safe, targeted and efficient CRISPR-based gene therapy in vivo.

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