• 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
  • Operator
  • Taipei Veterans General Hospital
  • Booth
  • 精準健康(生技&新藥) Precision Health(Biotech & New Drug Development)
  • Contact
  • 周士傑
  • Email
  • sjchou3@vghtpe.gov.tw
Technical Description 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.
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.