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
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
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