Schiffelers Lab

University Medical Center Utrecht

West-Nederland (NL3) · Netherlands

ERC-funded
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Research focus

ERC Advanced Grant · 2024

Advancing Gene Transfer Capabilities

Innovation: We recently developed a targeted lipid nanopar We recently developed a targeted lipid nanoparticle (tLNP) that can insert a gene directly into the genome of T cells in vivo, with efficiency comparable to viral methods. This innovation creates a unique opportunity to track gene delivery using barcoded synthetic vectors. These barcodes help us understand the key factors needed for effective DNA delivery and the biological consequences of gene transfer. Background: Gene therapy holds great promise for treating various diseases. Viral vectors are currently the most common method for gene transfer, but they come with limitations like size restrictions on nucleic acids, triggering…

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