Table 3.
Pathology | Therapeutic genes | IRES | Vector type | Outcome | Ref. |
Ischemic heart disease | VEGFA + FGF2 | EMCV | Plasmid | Moderate benefits | Kukula et al[90], 2011 |
Parkinson | TH + AADC + CH1 | EMCV | Lentivector | Benefits for 15/15 patients | Palfi et al[91], 2014 |
Mucopolysaccharidosis type IIIA | SGSH + SUMF1 | EMCV | Lentivector | Benefits for 1/4 patients, stabilization for 3/4 | Tardieu et al[71], 2014 |
TH: Tyrosine hydroxylase; AADC: Aromatic L-amino acid decarboxylase; CH1: GTP cyclohydrolase-1; SGSH: N-sulfoglycosamine sulfohydrolase; SUMF1: Sulfatase-modifying factor.