| Technical Name | Innovative Nanogene CorrectionCRISPR/Cas9 for the Treatment of Hereditary Retinal Diseases | ||
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| Project Operator | National Yang-Ming University | ||
| Project Host | 邱士華 | ||
| Summary | 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. |
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| Scientific Breakthrough | 1. Non-viralhighly biocompatible 2. Easy to metabolizecarry large size CRISPR / Cas9 components 3. Release methods expressed in cellsin vivoused for gene editing 4. Establishment of patient-specific iPSCin vitro functional 3D retina |
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| 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 |
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| Keyword | Hereditary disease Retina nanodiamond human induced pluripotent stem cells CRISPR Gene therapy Gene editing nanomedicine Non-viral vector Precision Medicine | ||
- shchiou@vghtpe.gov.tw
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