Table 2.

Therapeutic use of miRNAs and antagomirs in vivo

miRNA‡	Delivery method	Model used	Phenotypes	Refs
let-7	Intranasal delivery of viral particles	KrasG12D/+ autochthonous NSCLC mouse	Suppression of lung tumour initiation when delivered at the same time as transgene activation	145, 146
	Intratumoral injection of lipid-based mimetic	Subcutaneous H460 NSCLC xenografts	Interfered with further tumour growth	62
	Intranasal delivery of viral particles	KrasG12D/+ autochthonous NSCLC mouse	Reduced burden of tumours that were allowed to preform 10 weeks before let-7 therapy	62
	Systemic delivery of lipid-based mimetic	KrasG12D/+ autochthonous NSCLC mouse	Reduced burden of tumours that were allowed to preform 10 weeks before let-7 therapy	90
	Transfected into cells before transplantation	Subcutaneous human U251 or U87 glioblastoma cells	Reduced tumour formation	91
	Transduced into cells before transplantation	Chemotherapy-resistant breast tumour initiating cells	Reduced tumour formation and metastasis	92
miR-143 and miR-145	Transduced into cells before transplantation	Subcutaneous MiaPaCa2 and Panc1 PDAC xenografts	Unable to form tumours	94
	Systemic delivery of lipid-based expression vectors	Subcutaneous MiaPaCa2 PDAC xenografts	Inhibited growth	96
	Systemic delivery of lipid-based expression vectors	Orthotopic MiaPaCa2 PDAC xenografts	Inhibited growth	96
miR-143	Systemic delivery of anti-miR-143	p21-HBx HCC mouse	Inhibited primary tumour and local and distant metastatic growth	100
miR-34	Intratumoral delivery of lipid-based mimetic	Subcutaneous H460 NSCLC xenografts	Inhibited growth	106
	Systemic delivery of lipid-based mimetic	Subcutaneous H460 and A549 NSCLC xenografts	Inhibited growth	106
	Systemic delivery of lipid-based mimetic	KrasG12D/+ autochthonous NSCLC mouse	Reduced burden of tumours that were allowed to preform 10 weeks before miR-34 therapy	90
	Transfected oligonucleotides into cells before transplantation	Subcutaneous prostate cancer xenografts (multiple cell lines)	Reduced tumour incidence	107
	Transduced cells with virus encoding precursor (pre)-miR-34 before transplantation	Subcutaneous prostate cancer xenografts (multiple cell lines)	Reduced tumour incidence	107
	Intratumoral injection of lipid-based mimetic	Subcutaneous PPC1 prostate cancer xenografts	Inhibited growth	107
	Systemic delivery of lipid-based mimetic	Orthotopic PC3 prostate cancer xenografts	Reduced tumour burden	107
	Systemic delivery of lipid-based mimetic	Orthotopic LAPC9 prostate cancer xenografts	Reduced lung metastasis without affecting primary tumour growth; extended survival	107
	Systemic delivery of lipid-based expression vectors	Subcutaneous and orthotopic MiaPaCa2 PDAC xenografts	Inhibited growth	96
miR-122	Transduced into cells before transplantation	Orthotopic SKHEP1 HCC xenografts	Reduced tumorigenesis, angiogenesis and intrahepatic metastasis	111
	Systemic delivery of nucleic acid antagomir-miR-122	HCV-infected non-human primates	Suppression of HCV viraemia and improved liver pathology	112
	Systemic delivery of lysine–lipid nanoparticle antagomir-miR-122	C57BL/6J mice	Decreased plasma cholesterol levels	113
	Systemic delivery of antagomir-miR-122	C57BL/6J mice	Decreased plasma cholesterol levels	118
miR-26a	Systemic delivery of adeno-associated virus	HCC liver cancer model: MYC driven by the liver activator promoter	Inhibition of proliferation; induction of apoptosis; disease protection	114
miR-10b	Systemic delivery of antagomir-miR-10b	Implantation of 4T1 breast cancer cells into the mammary fat pad	Prevented metastasis with no effect on the primary tumour	84

^‡miRNAs are presented in the order in which they are discussed in the main text. miRNAs for which there is therapeutic evidence in endogenously occurring tumours or orthotopic implants are included. In some instances xenograft models are included but only as supporting evidence.

HCC, hepatocellular carcinoma; HCV, hepatitis C virus; miRNA, microRNA; NSCLC, non-small-cell lung cancer; PDAC, pancreatic ductal adenocarcinoma.