An intriguing dream of chemists, molecular biologists and clinicians alike is the ability to address and treat genetically inherited diseases or diseases caused by genetic mutations. Real treatment would require personalized gene editing to convert disease causing gene mutations back to fully functional genes. A new and revolutionizing technology called CRISPR/Cas has recently been established and has changed molecular biology and the therapy outlook for inherited diseases forever. The CRISPR/Cas technology could, despite current limitations, if selective, personalized, and effective in different biological environments, change society fundamentally. By far the most challenging aspect for translation of the full potential of CRISPR/Cas into clinical protocols is the efficient and targeted delivery of CRISPR/Cas gene editing tools to selected tissues and organs with the ultimate challenge being the brain. This project engages in the development of a highly flexible and non-viral delivery platform based 1/12 R346-2020-1853 on smart lipid nanocarriers. All components are designed to be surface anchored and to selfassemble by a simple controlled mixing process for all components with the smart nanocarrier into an active and effective CRISP/Cas delivery vehicle for the treatment of genetically inherited diseases or diseases caused by genetic mutations.
|Effective start/end date
|01/01/2021 → 31/12/2022