Table 1.

A synoptic view of current issues in gene and cell therapy

Challenge	In vivo gene therapy	Ex vivo gene therapy	Cell therapy
Vector of choice	Adeno-associated vector	Lentivector	—
Cell of choice	—	Satellite cells	Satellite cells Mesoangioblasts
		Mesoangioblasts	Induced pluripotent stem cells
		Induced pluripotent stem cells
Delivery route	Intramuscular	Intramuscular	Intramuscular
	Intravenous	Intra-arterial	Intra-arterial
	Intravenous loco-regional	Intravenous	Intravenous
Gene correction	Replacement	Replacement	Replacement
	Exon skipping	Exon skipping
	Genome editing	Genome editing
Immune response	Wild-type gene product	Wild-type gene product	Wild-type gene product
	Therapeutic protein (e.g., nuclease)	Therapeutic protein (e.g., nuclease)
	Vector (capsid)

The table offers a simple overview of current major issues in the field.