Table 1.

Preclinical data providing evidence for in vivo CAR T cell generation

Vector			Mouse model		CAR		Reference
Platform	Targeted receptor	Dosing and routeb	Straind	Immune transplantse	Configurationf	Target cellsg
LV with NiV glycoproteins	Human CD8	2 × 106 TU (i.p.)	NSG	Activated human PBMCs (i.p.)	hCD19-28ζ-CAR	Raji (i.p.)	Pfeiffer et al.19
		2 × 106 TU (i.v.)	NSG	Human CD34+ CB cells	hCD19-28ζ-CAR	Endogenous B cells
LV with NiV glycoproteins	Human CD8	2.5 × 1011 particles (i.v.)	NSG	Activated human PBMCs (i.v.)	hCD19-28ζ-CAR	Nalm-6 luc, (i.v.)	Agarwal et al.20
LV with MV glycoproteins	Human CD4	4 × 1010 particles (i.p.)	NSG	Activated human PBMCs (i.p.)	hCD19-28ζ-CAR	Endogenous B cells	Agarwal et al.21
		1 × 1011 particles (i.v.)	NSG	Activated human PBMCs (i.v.)	hCD19-28ζ-CAR	Nalm-6 luc, (i.v.)
		4 × 1010 particles (i.v.)	NSG	Human CD34+ CB cells	hCD19-28ζ-CAR	Endogenous B cells
LV with NiV glycoproteins	Human CD3	2 × 1011 particles (i.v.)	NSG	Human CD34+ CB cells	hCD19-28ζ-CAR	Endogenous B cells	Frank et al.22
LV with SINV glycoproteins	Human CD3	5 × 1010 particles (i.v.)	NSG	Activated human PBMCs (i.v.)	hCD19-28ζ-CAR	BV-173 luc (i.v.)	Huckaby et al.23
AAV-DJ	N/Aa	1 × 1011 vg (i.p.)	NCG	Human PBMCs (i.p.)	hCD4-28-4-1BBζ-CAR	Endogenous CD4 T cells	Nawaz et al.24
		2 × 1011 vg (i.p.)	NCG	Human PBMCs (i.p.)	hCD4-28-4-1BBζ-CAR	MT-2 ATL (i.p.)
NC with DNA	Mouse CD3	3 × 1011 particles/day on five consecutive days (i.v.c)	Albino B6	N/A	mCD19-4-1BBζ-CAR	N/A	Smith et al.25
		3 × 1011 particles/day on five consecutive days (i.v.c)	Albino B6	N/A	mCD19-4-1BBζ-CAR	Eμ-ALL(i.v.)
NC with mRNA	Human CD8	6 weekly doses; 50 μg mRNA/dose (i.v.)	NSG	Human T cells (i.v.)	hCD19-28ζ-CAR	Raji-luc (i.v.)	Parayath et al.26
		6 weekly doses; 50 μg mRNA/dose (i.v.)	NSG	Human T cells (i.v.)	hROR1-4-1BBζ-CAR	LNCaP C42 prostate cancer cells (orthotopic)
	Human CD3	4 weekly bursts of 3 daily doses; 15 μg mRNA/dose (i.v.)	B6	N/A	mCD19-28ζ-CAR	Eμ-ALL (i.v.)
LNP with mRNA	Mouse CD5	Single dose of 10 μg LNPs (i.v.)	B6	N/A	mFAP-28ζ-CAR	Endogenous fibrotic cardiac fibroblast	Rurik et al.27

^aN/A, not applicable.

^bTU, transducing unit; i.p., intraperitoneal injection; i.v., intravenous injection; vg, vector genome.

^cover 20 min with infusion pump.

^dNSG, NOD.Cg.Prkdc^scidIL2rg^tmWjl/SzJ; NCG, NOD/ShiLtJGpt-Prkdc^em26Cd52Il2rg^em26Cd22/Gpt; Albino B6, C57BL/6J-Tyr^c−2J/J; B6, C57BL/6J.

^ePBMCs, human peripheral blood mononuclear cells; CB, cord blood-derived.

^fh, anti-human; m, anti-mouse.

^gluc, luciferase expressing.