DUAL AAV VECTOR SYSTEM FOR CRISPR/CAS9 MEDIATED CORRECTION OF HUMAN DISEASE

摘要

A dual vector expression system is provided for delivery of CRISPR/Cas9, sgRNA and donor templates for supplying corrected genes or gene sequences to neonatal subjects. An AAV8 system for delivery to proliferating cells such as hepatocytes is described, as are other dual AAV vector systems for targeting to other cells.