Technical Name |
Innovative Nanogene Correction and CRISPR/Cas9 for the Treatment of Hereditary Retinal Diseases |
Project Operator |
National Yang-Ming University |
Project Host |
邱士華 |
Summary |
NDs carrying the RS1 gene-editing platform can enter human iPSC and perform gene editing; animal experiments have also shown that this platform can enter the retina and edit the RS1 gene, causing changes in the retina. In addition, the establishment of XLRS patient-PSCs and in vitro "functional 3D retinal" differentiation technology can be used as a cell model platform to verify. |
Scientific Breakthrough |
1. Non-viral and highly biocompatible 2. Easy to metabolize and carry large size CRISPR / Cas9 components 3. Release methods expressed in cells or in vivo and used for gene editing 4. Establishment of patient-specific iPSC and in vitro functional 3D retina |
Industrial Applicability |
Our technology is curable and highly bio-safe and can be standardized and produced in large quantities. Gene therapy will have a breakthrough impact. Effective therapeutic drugs will be developed for hereditary diseases, and 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 |
Keyword |
Hereditary disease Retina nanodiamond human induced pluripotent stem cells CRISPR Gene therapy Gene editing nanomedicine Non-viral vector Precision Medicine |