• Technical Name
  • Innovative Nanogene CorrectionCRISPR/Cas9 for the Treatment of Hereditary Retinal Diseases
  • Operator
  • National Yang-Ming University
  • Booth
  • Online display only
  • Contact
  • 邱士華
  • Email
  • shchiou@vghtpe.gov.tw
Technical Description NDs carrying the RS1 gene-editing platform can enter human iPSCperform gene editing animal experiments have also shown that this platform can enter the retinaedit the RS1 gene, causing changes in the retina. In addition, the establishment of XLRS patient-PSCsin vitro \"functional 3D retinal\" differentiation technology can be used as a cell model platform to verify.
Scientific Breakthrough 1. Non-viralhighly biocompatible2. Easy to metabolizecarry large size CRISPR / Cas9 components3. Release methods expressed in cellsin vivoused for gene editing4. Establishment of patient-specific iPSCin vitro functional 3D retina
Industrial Applicability Our technology is curablehighly bio-safecan be standardizedproduced in large quantities. Gene therapy will have a breakthrough impact. Effective therapeutic drugs will be developed for hereditary diseases,iPSC will be used as a testing platform for different diseases. In addition, cooperation will be carried out to enhance the technology of gene editing therapy