NANOPARTICLES-MEDIATED CRISPR-CAS9 FOR GENE THERAPY
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Issued Date -
May 23, 2024
app pub date -
Nov 20, 2023
filing date -
Nov 18, 2022
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Abstract
The present invention is directed to an integrated conceptual strategy for a gene delivery system, using the combination of nanoparticles, CRISPR-Cas9, and the HITI strategy to deliver CRISPR-Cas9 and achieve effective genome editing; wherein the advanced nanoparticles to overcome the limited packaging size of AAV-based vehicles. Also provided is a promising therapeutic solution for the treatment of hereditary diseases via gene therapy
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