Fig. 17

Schematic diagram of basics of rAAV-gene therapy. Left: The gene of interest is packaged within a rAAV vector. When the virus infects the host cell, it injects its DNA-containing gene of interest. This foreign DNA then crosses the nuclear membrane and binds with host DNA. Using protein machinery, the nucleus can make DNA and protein using the inserted DNA, replacing mutated or abnormal genes from host cell. Right: CRISPR-Cas9 system can be used to correct defect gene in PD and other genetic diseases. In presence of guide RNA (g-RNA) CRISPR-Cas9 enzyme can breakdown the DNA double strands in the locus where mutated or faulty genes are located. Then using DNA repair system, the normal DNA can be inserted in the cut site to get normal gene expression