Table 1.

Reported genotoxic events in HSC gene therapy clinical trials

Diseases	Patients	Vector backbone	Transgene	Follow-up	Severe adverse effects		Insertion sites (clonal dominance)	Clinical outcome	References
SCID-X1	9	MFG	γC	9 years	T-ALL	No.1	LMO2	Died despite chemotherapy	[1,2,10,15]
		γ-retroviral		(range, 8 to 11)	in four patients	No.2	LMO2	Remission with chemotherapy
		vector				No.3	CCND2	Remission with chemotherapy
						No.4	LMO2, BMI1	Remission with chemotherapy
						Other 5	No clonal dominance	Alive, improved immune function
SCID-X1	10	MFG	γC	5 years	T-ALL	No.1	LMO2	Remission with chemotherapy	[14,16]
		γ-retroviral			in one patient	Other 9	No clonal dominance	Alive with improved immune
		vector						function
ADA-SCID	10	GIADAl	ADA	4.0 years	None		Integration hotspots near	All alive with improved	[3,4]
		MLV-based		(range,			DYRK1A,BLM,LMO2,	immune and metabolic
		retroviral vector		1.8 to 8.0)			CCND2 and BCL2 no clonal	parameters
							selection )
X-CGD	2	pSF7, SFFV-	gp91phox	4 years	MDS	No.1	MDS1/Evi1, PRDM16, and	Died from sepsis	[5,9]
		based retrovirus					SETBP1	27 months
		vector			MDS	No.2	MDS1/Evi1, PRDM16, and	Underwent an allogeneic
							SETBP1	HSC transplantation at month 45
X-CGD	3	MFGS	gp91phox	3 years	None		No clonal dominance	Clinical improvement	[6]
		retroviral vector
X-ALD	2	HIV-1-derived	ABCD1	24 to 30 months	None		No clonal dominance	Alive with decreased progression	[7]
		lentiviral vector						of neurologic phenotype
β-thalassemia	1	HIV-1-derived	β-globin	33 months	None		Dominant HMGA2 expression	Alive and transfusion-	[26]
		lentiviral vector					clone	independent
WAS	2	CMMP	WASP	3 years	T-ALL in one		LMO2, CCND2, and BMI1	T-ALL patient with ongoing	[8]
		retroviral vector			patient(LMO2)		in T-cells, and MDS1	chemotherapy, the other alive
							/EVI1, PRDM16, and	with improved immune
							SETBP1 in granulocytes	function and decreased cytopenias