Technical Name 結合奈米技術與CRISPR/Cas9基因編輯應用於治療遺傳性疾病
Project Operator Taipei Veterans General Hospital
Project Host 周士傑
Supramolecular nanoparticles is a convenient, flexible,modular synthetic non-viral gene delivery vehicle for drug/gene delivery. The self-assembly of SMNPs is enabled upon mixing three different molecular building blocks, namely CD-PEI, Ad-PAMAM,Ad-PEG via Ad/CD motif-based molecular recognition. This allows modular control over the sizes, surface chemistry,payloads of SMNP vectors, thus holding promise for diverse therapeutic applications, especially for developing nanoparticle-based gene delivery platforms.
Technical Film
Scientific Breakthrough
CRISPR-Cas9 is the latest gene-editing technology, providing a convenientstraightforward technique for gene-editing. However, there are still lots challenges before it can be applied to the clinical. We conducted the nanoparticles to encapsulate the CRISPR/Cas9 gene-editing material into the animals’ eyes,in blood cells to achieve the therapeutic effect. In sum, this technology successfully combines nanotechnologygene-editing technology to provide revolutionary general new ideasestablish new treatment models for bloodeye genetic diseases.
Industrial Applicability
The gene therapy technology has proven its efficacy in both bloodanimals,has a high degree of biological safety,can be mass-produced in a standardized manner. Gene therapy will have a breakthrough impact. In addition, collaborations will be launched to enhance the technology of gene editing therapy. Further development will be used for the treatment of more genetic diseases.
Matching Needs
Keyword Nanoparticle Nanowire Sickle Cell Disease Beta-Thalassemia XLRS CRISPR/Cas9 HBB RS1 Gene editing
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