Table 1.

HIV Gene Therapy Clinical Trials

Gene	Vector	Target Cell	Phase	Status	Reference or ClinicalTrials.gov Trial
RNA-Based Approaches
U5 ribozyme and pol ribozyme	GV	CD4	I	completed	203
tat and tat/rev ribozyme	GV	CD34	I	completed	38
tat/vpr ribozyme (Rz2/Oz1)	GV	CD4	I	completed	204, 205
	GV	CD34	I	completed	206
	GV	CD34	II	completed	75
RRE decoy RNA	GV	CD34	I	completed	40
env antisense RNA (VRX-496)	LV	CD4	I	completed	44
	LV	CD4	II	active	53
Protein-Based Approaches
Dominant-negative Rev (RevM10)	GP	CD4	I and II	completed	41
	GV	CD4	I and II	completed	42
	GV	CD34	I	completed	43
Dominant-negative Rev (TdRev)	GV	CD34	I	completed	207
Membrane-anchored fusion inhibitor (maC46)	GV	CD4	I	completed	56
CXCR4-anchored fusion inhibitor (C34-CXCR4)	LV	CD4	I	recruiting	NCT03020524
CCR5 CRISPR/Cas9	NA	CD34	NA	recruiting	NCT03164135
CCR5 zinc-finger nuclease (SB-728)	Ad	CD4	I	completed	63
	Ad	CD4	I	completed	NCT01044654a
	Ad	CD4	I and II	completed	NCT01252641b
	Ad	CD4	I and II	completed	NCT01543152c
	mRNA	CD4	I	active	NCT02388594
	mRNA	CD4	I and II	active	NCT02225665
	mRNA	CD34	I	recruiting	NCT02500849
MazF endoribonuclease	LV	CD4	I	active	NCT01787994
Combination Approaches
TAR antisense RNA, dominant-negative Rev	GV	CD4	I	completed	208
CCR5 shRNA, TRIM5α, TAR decoy RNA	LV	CD34	I and II	recruiting	NCT02797470
tat/rev shRNA, TAR decoy RNA, CCR5 ribozyme	LV	CD34	I	completed	39
(rHIV7-shI-TAR-CCR5RZ)	LV	CD34	I	active	NCT01961063
	LV	CD34	I	recruiting	NCT02337985
CCR5 shRNA, maC46 (Cal-1)	LV	CD4 CD34	I and II	completed	NCT01734850
Cal-1 plus MGMT(P140K)	LV	CD34+	I	recruiting	NCT02343666

GV, gammaretroviral vector; LV, lentiviral vector; Ad, adenoviral vector; GP, gold particles; CD4, CD4⁺ T cells; CD34, CD34⁺ HSPCs.

^aT cell dose escalation, patients with ART failure, and patients with heterozygote CCR5 delta-32 mutation.

^bPatients with ART failure.

^cCyclosphosphamide dose escalation.