Description
Genome editing and repair by using the CRISPR/Cas9 system and non-viral gene therapy techniques of the Z mutation of the SERPINA1 gene that encodes for the alpha-1 antitrypsin gene in monocytes and hepatocytes of patients with alpha-1 antitrypsin deficiency.
Keywords
gene therapy, CRISPR-Cas9, AATD
Manager UV
- Dasi Fernandez, Francisco Jose
- PDI-Titular d'Universitat
Frascati classification
