CORDIS Project
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The POLYMORPH project focuses on improving gene therapy by developing nanoparticle carriers that can efficiently deliver therapeutic nucleic acids to specific tissues, avoiding liver accumulation. This innovative approach aims to enhance the effectiveness of treatments for rare genetic disorders through better targetin…
Gene therapy holds the potential to be an enabling technology to truly cure (rare) diseases by directly correcting errors in the patient’s genetic database underlying the disorder.
For these corrections therapeutic nucleic acids (NAs) are typically used.
However, the precise and efficient delivery of these NAs remains a critical bottleneck, hindering the full-scale clinical implementation of gene therapy. NAs are large, highly charged and unstable molecules, hampering transport and passing (intr…
UNIVERSITEIT UTRECHT
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