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. Author manuscript; available in PMC: 2020 Nov 14.
Published in final edited form as: Cell. 2019 Nov 14;179(5):1033–1055. doi: 10.1016/j.cell.2019.10.017

Table 1.

Oncogenic or tumor suppressive non-coding RNAs with in vivo experimental evidence

Name ncRNA Cancer types examined In vivo experimental techniques used Cancer-related mechanisms and/or functions of ncRNA References
Oncogenic ncRNAs

miR-10b miRNA breast, glioblastoma antimiRs, CRISPR-Cas9 knockdown in mouse xenografts and allografts, transgenic knockout mouse models targets several transcripts that encode regulators of cell cycle progression, migration, invasion, and metastasis Ma et al., 2010, Kim et al., 2016, El Fatimy et al., 2017
miR-21 miRNA lung, B cell lymphoma transgenic knockout, overexpression mouse models targets transcripts that encode negative regulators of RAS signaling, leading to increased proliferation and decreased apoptosis Hatley et al., 2010, Medina et al., 2010
miR-31 miRNA lung, breast overexpression in mouse xenografts, transgenic knockout, overexpression mouse models targets transcripts that encode regulators of RAS, WNT, and TGFβ signaling to increase proliferation, stem cell renewal, and metastasis Edmonds et al., 2016, Lv et al., 2017
miR-155 miRNA lymphoma transgenic overexpression mouse model, treatment with antimiRs targets SHIP1 transcript, a negative regulator of AKT, to increase proliferation and survival O’Connell et al., 2009, Babar et al., 2012, Cheng et al., 2015
miR-221 miRNA liver overexpression, treatment with antimiRs in mouse xenografts targets transcripts of tumor suppressors and cell cycle inhibitors (e.g., p27, PTEN) to increase proliferation and decrease apoptosis Pineau et al., 2010, Park et al., 2011
LeuCAG3’tsRNA tsRNA liver LNA knockdown in PDX enhances translation of transcripts encoding ribosomal proteins, leading to increased ribosome biogenesis and proliferation Kim et al., 2017
ARLNC1 lncRNA prostate shRNA or ASO knockdown in mouse xenografts interacts with the mRNA encoding AR, a nuclear receptor, to promote oncogenic AR signaling, proliferation, and suraval Zhang et al., 2018
CamK-A lncRNA breast shRNA, siRNA knockdown in mouse xenografts/PDX interacts with and controls activity of kinases that modulate calcium-induced NF-KB signaling, leading to remodeling of the tumor microenvironment Sang et al., 2018
CCAT1 lncRNA colorectal, esophageal shRNA, siRNA knockdown in mouse xenografts interacts with transcription factors (e.g., SOX2, p63) to activate expression of genes involved in increasing proliferation and decreasing apoptosis Kim et al., 2014, Jiang et al., 2018
CTBP1-AS lncRNA prostate siRNA knockdown, overexpression in mouse xenografts recruits chromatin modifying, splicing factors to promoter of a nuclear receptor corepressor (CTBP) to decrease its expression, leading to increased oncogenic AR activity Takayama et al., 2013
DSCAM-AS1 lncRNA breast shRNA knockdown in mouse xenografts interacts with proteins of the hnRNP complex involved in RNA processing and mediates proliferation, invasion, and metastasis Niknafs et al., 2016
EPIC1 lncRNA breast shRNA knockdown in mouse xenografts interacts with MYC transcription factor and increases its activation of target genes, leading to enhanced cell cycle progression Wang et al., 2018
FAL1 lncRNA ovarian breast shRNA, siRNA knockdown in mouse xenografts stabilizes components of PRC1 chromatin modifying complex to mediate expression of genes involved in proliferation and suraval Hu et al., 2014
HOTAIR lncRNA breast siRNA knockdown, overexpression in mouse xenografts recruits PRC2, LSD1/CoREST/REST chromatin modifying complexes, scaffolds transcription factors at target promoters of genes involved in invasion, metastasis, and proliferation Gupta et al., 2010, Li et al., 2016b
LINK-A lncRNA breast shRNA knockdown in mouse xenografts, transgenic overexpression in mammary gland, LNA knockdown interacts with kinases that control HIF1α activity, glycolysis, enhances degradation of tumor suppressors (RB, p53) and antigen peptide-loading complexes to promote immune evasion Lin et al., 2016, Hu et al., 2019
lncARSR lncRNA RCC shRNA knockdown, overexpression in mouse xenografts interacts with transcriptional coactivator YAP and acts as a ceRNA for miRNAs that target RTK transcripts, leading to enhanced suraval and propagation of tumor-initiating cells Qu et al., 2016a, Qu et al., 2016b
PCAT-1 lncRNA prostate overexpression in mouse xenografts represses expression of BRCA2 tumor suppressor to impact DNA damage repair Prensner et al., 2014a
PVT1 lncRNA colorectal, gastric CRISPR-Cas9 or shRNA knockdown, overexpression in mouse xenografts activates oncogenic signaling (MYC, STAT3) and represses expression of tumor suppressors (p15, p16), resulting in increased proliferation, angiogenesis and decreased apoptosis Tseng et al., 2014, Kong et al., 2015, Zhao et al., 2018
SAMMSON lncRNA melanoma GapmeR knockdown in PDX interacts with and controls subcellular localization of proteins that regulate mitochondrial homeostasis and metabolism Leucci et al., 2016
SChLAP1 lncRNA prostate shRNA knockdown in mouse xenografts interacts with and antagonizes activity of the SWI/SNF chromatin modifying complex to promote invasion and metastasis Prensner et al., 2013
THOR lncRNA lung, melanoma CRISPR-Cas9 knockdown, overexpression in mouse xenografts, transgenic knockout, overexpression in zebrafish binds IGF2BP1 to stabilize interactions with oncogenic target mRNAs, in turn stabilizing those transcripts and promoting proliferation Hosono et al., 2017
BRAFP1 pseudogene B cell lymphoma transgenic overexpression mouse model acts as a ceRNA for miRNAs that target the BRAF transcript, leading to increased BRAF expression, MAPK signaling, and proliferation Karreth et al., 2015
circCCDC66 circRNA colorectal siRNA knockdown in mouse xenografts sponges several miRNAs that target oncogenic transcripts (e.g., MYC), promoting proliferation, migration, and invasion Hsiao et al., 2017
circCTNNB1 circRNA gastric shRNA knockdown, overexpression in mouse xenografts binds a DEAD-box RNA helicase (DDX3) to increase transcription factor (YY1) activation of WNT/p-catenin target genes Yang et al., 2019

Tumor suppressive ncRNAs

let-7 miRNA lung antimiRs, mimetics in mouse xenografts or transgenic lung cancer mouse models targets several transcripts that encode oncogenes, including RAS, leading to decreased cell cycle progression and proliferation Trang et al., 2010, Trang et al., 2011
miR-15a/16-1 miRNA prostate, leukemia knockdown, overexpression in mouse xenografts, transgenic knockout mouse targets several transcripts that encode cyclins, CDKs, and anti-apoptotic proteins, thereby increasing apoptosis and inhibiting proliferation Bonci et al., 2008, Klein et al., 2010
miR-34a miRNA lung, prostate, breast overexpression, mimetics, antimiRs in mouse xenografts, mimetics in transgenic lung cancer mouse models targets several oncogenic transcripts encoding cyclins, CDKs, cell adhesion molecules, RTKs, and other non-RTKs, resulting in decreased proliferation, invasion, and suraval Liu et al., 2011, Trang et al., 2011, Kasinski et al., 2012, Adams et al., 2016b
miR-122 miRNA liver transgenic knockout mouse models, overexpression in transgenic liver cancer mouse models targets expression of several genes involved in lipid metabolism, proliferation, and inflammation Hsu et al., 2012, Tsai et al., 2012
miR-506 miRNA ovanan mimetics in mouse xenografts targets SNAI2 transcript to decrease its expression and inhibit migration, invasion, and EMT Yang et al., 2013a
GAS5 lncRNA glioblastoma overexpression in mouse xenografts acts as a nuclear receptor response element mimic for GR, decreases oncogenic miRNA expression, leading to enhanced apoptosis, decreased proliferation, invasion, and migration Zhang et al., 2013, Zhao et al., 2015
LET lncRNA liver, colorectal shRNA knockdown, overexpression in mouse xenografts interacts with and destabilizes a dsRNA binding protein (NF90), a key factor involved in regulation of HIF-1a levels and cell invasion Yang et al., 2013b
MEG3 lncRNA lung overexpression in mouse xenografts increases p53 tumor suppressor levels to activate apoptosis and decrease proliferation Lu et al., 2013
PTENP1 pseudogene RCC, bladder overexpression in mouse xenografts acts as a ceRNA for miRNAs that target the PTEN tumor suppressor transcript, leading to increased apoptosis, decreased proliferation, migration, and invasion Yu et al., 2014, Zheng et al., 2018
circHIPK3 circRNA bladder overexpression in mouse xenografts sponges miRNAs to suppress expression of an endoglycosidase (HPSE), thereby decreasing levels of pro-migratory and angiogenic factors Li et al., 2017

Context-dependent ncRNAs

miR-26a miRNA glioma, leukemia, liver, colorectal overexpression in transgenic glioma, liver cancer mouse models, transgenic overexpression mouse models targets both tumor suppressor (PTEN) and cyclin (CCND2/E2) transcripts to either increase or decrease proliferation, depending on the context Huse et al., 2009, Kota et al., 2009, Mavrakis et al., 2011, Zeitels et al., 2014
miR-29 miRNA leukemia, glioblastoma mimetics in mouse xenografts, transgenic overexpression mouse models promotes proliferation in B cells but can also target and decrease expression of transcipts that enhance lipid synthesis (SCAP, SREBP-1), proliferation (CDK6), and apoptosis (MCL-1) Garzon et al., 2009, Santanam et al., 2010, Ru et al., 2016
MALAT1 lncRNA lung, breast genetic knockout, overexpression in mouse xenografts and allografts, transgenic knockout, overexpression mouse models, ASOs in mouse xenografts or transgenic models promotes expression of genes involved in metastasis, conversely, shown to also inhibit metastasis through interaction with/inhibition of a pro-metastatic transcription factor (TEAD) Gutschner et al., 2013, Arun et al., 2016, Kim et al., 2018
NEAT1 lncRNA prostate, skin, pancreatic shRNA knockdown, overexpression in mouse xenografts, transgenic knockout mouse models mediates oncogenic nuclear receptor (ER) signaling, prevents DNA damage and activation of p53 tumor suppressor, leading to proliferation, invasion, and decreased apoptosis, conversely, shown to also prevent transformation and proliferation in other settings Chakravarty et al., 2014, Adriaens et al., 2016, Mello et al., 2017
NKILA lncRNA breast shRNA knockdown, overexpression in mouse xenografts/PDX binds/inhibits NF-KB and downstream inflammation, which increases apoptosis, reduces invasion, promotes activation-induced cell death in CTLs, T>1 cells, leading to immune evasion Liu et al., 2015, Huang et al., 2018

Abbreviations: AR, androgen receptor, ASO, antisense oligonucleotide, CDK, cyclin-dependent kinase, ceRNA, competitive endogenous RNA, circRNA, circular RNA, CTL, cytotoxic T lymphocyte, EMT, epithelial-to-mesenchymal transition, ER, estrogen receptor, GR, glucocorticoid receptor, hnRNP, heterogeneous nuclear ribonucleoprotein, LNA, locked nucleic acid, lncRNA, long non-coding RNA, miRNA, microRNA, ncRNA, non-coding RNA, PDX, patient-derived xenograft, RCC, renal cell carcinoma, RTK, receptor tyrosine kinase, tsRNA, tRNA-derived small RNA