Table 1.

Cancer Mouse models developed by CRISPR-Cas9 based genome editing

In vivo
Cancer type	Mouse strain and genotype	Delivery	Mutations/genomic alterations	References
Lung adenocarcinoma	CD1 and C57BL/6J (B6)	adenoviral	Eml4-Alk translocation in lung	Maddalo D et al, Nature 2014
Lung adenocarcinoma	p53+/− or p53−/−	lentiviral	Eml4-Alk translocation in lung	Blasco RB et al, Cell Reports 2014
Lung adenocarcinoma	KrasLSL-G12D/+	lentiviral	Nkx, Pten, Apc	Sanchez-Rivera FJ et al, Nature 2014
Liver cancer	FVB/NJ mice	hydrodynamic injection	p53, Pten,Ctnb1	Xue W et al, Nature 2014
Burkitt Lymphoma	Arf−/− Eμ-Myc	lentiviral and retroviral	p53	Malina et al, Genes and Dev 2013
Pancreatic ductal adenocarcinoma	KrasLSL-G12D/+;R26LSL-Tom;H11LSL-Cas9/+	retrograde ductal injection of adeno/lentivirus	Lkb1	Chiou et al, Genes and Dev 2015
Pancreatic ductal adenocarcinoma	Kras+/L5L-G12D;Trp53loxP/loxP	lentiviral	p53, Kras G12D, and p57	Mazur et al, Nature Medicine 2015
medulloblastoma	C57BL/6N mice	PEI-mediated transfection andin utero electroporation	Ptch1	Zuckermann M, et al Nature Communications 2015
Glioblastoma	Crl:CD1(ICR) mice	PEI-mediated transfection andin utero electroporation	Trp53, Pten, Nf1	Zuckermann M, et al Nature Communications 2015
Ex vivo
Cancer type	Mouse strain and genotype	Delivery	Mutations/genomic alterations	References
Acute Myeloid Leukemia	C57Bl/6 mice or heterozygous Flt3-ITD knock-in mice	lentiviral	Dnmt3a, Ezh2,Runx1, Smc3 and Nf1	Heckl et al, Nature Biotech 2014
Acute Myeloid Leukemia	p53 null HSPC	lentiviral	Mll3	Chen et al, Cancer Cell 2014
Burkitt Lymphoma	HSPC from Eμ-Myc mice	lentiviral	p53	Aubrey et al, Cell Reports 2015
Xenograft
Cancer type	Mouse strain and genotype	Delivery	Mutations/genomic alterations	References
Tumor regression	Nude-Foxn1nu mice	lipid transfection	Correction of PKC6 A509T	Antal C et al, Cell 2015
Cas9 knock-in
Cancer type	Mouse strain and genotype	Delivery	Mutations/genomic alterations	References
Lung adenocarcinoma	Cre-dependent Cas9	adenoviral delivery of guide RNA	p53, Lkb1 and Kras G12D	Platt et al, Cell 2014
Intestine hyperplastic polyps	doxycycline inducible Cas9	ESC targeting	Apc, Trp53	Dow et al, Nature Biotech 2015
Pancreatic ductal adenocarcinoma	KrasLSL-G12D/+;R26LSL-Tom;H11LSL-Cas9/+	retrograde ductal injection of adeno/lentivirus	Lkb1	Chiou et al, Genes and Dev 2015
In vivo metastases screen
Cancer type	Mouse strain and genotype	Delivery	Mutations/genomic alterations	References
Lung Metastases	KrasG12D/+;p53−/−;Dicer1+/−	lentiviral	Multiple hits from screen	Chen et al, Cell 2015