CORDIS Project
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This project develops advanced non-viral delivery systems using modified nanoparticles for precise genome editing, focusing on treating Sickle Cell Disease. By enhancing the efficiency of prime editors, it aims to simplify and improve the safety of gene therapy, making it more accessible for various genetic disorders.
Genome editing technologies based on CRISPR/Cas systems allow targeted genomic modification with unprecedented precision and have emerged as powerful alternatives to the conventional gene therapy approaches for various human diseases, with a series of clinical trials in progress.
However, some crucial challenges remain to be addressed to enhance efficiency and safety and decrease costs of treatments.
Current viral-based delivery systems are associated with high risk of toxicity and immunogenicit…
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
Netherlands, Rotterdam
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Spain, SANT FELIU DE LLOBREGAT
Type: SME
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: Yes
France, PARIS 15
Type: Research institute
Activity type: Research Organisations
SME: No
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