Internal ribosome entry site-based vectors for combined gene therapy

Table 3 Clinical studies of combined gene therapy

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.