Table I.

Knockout or mutant mouse models and their phenotypes according to stages of lymphatic vascular morphogenesis

Gene symbol Function Lymphatic vascular phenotype Expression pattern 
LEC differentiation 
Prox1 Transcription factor No LECs (−/−), chylous ascites (+/−); loss of LEC identity, chylous ascites, obesity (Prox1f/f; Tie2-Cre; Wigle et al., 1999; Harvey et al., 2005; Johnson et al., 2008) LECs, hepatocytes, lens fiber cells, pancreatic, lung, and intestinal endocrine cells, skeletal muscle, cardiomyocytes 
Sox18 Transcription factor No LECs(−/−, on C57BL/6J background); abnormal patterning (+/−); edema and chylous ascites (heterozygous ragged mutants; Pennisi et al., 2000; François et al., 2008) Endothelial cells 
Coup-TFII (NR2F2Transcription factor No LECs (−/−, deletion at or before E9.5); edema, loss of LEC identity, and sprouting (−/−, deletion at later stages; Lin et al., 2010; Srinivasan et al., 2010) Endothelial cells and SMCs 
Lymphangiogenesis 
Vegf-c–Vegfr-3 pathway 
Vegfr3 Receptor tyrosine kinase Hypoplasia, chylous ascites (+/Chy, ENU-induced mutation, loss of tyrosine kinase activity; Karkkainen et al., 2001); lymph sacs formed and defective sprouting (homozygous deletion of ligand-binding domain; Zhang et al., 2010) LECs, fenestrated blood vascular endothelial cells, blood vascular endothelial cells in tumors and during early embryogenesis 
Vegfc Growth factor, ligand for Vegfr-3 No sprouting of LECs from veins (−/−); hypoplasia, chylous ascites (+/−; Karkkainen et al., 2003) Macrophages, SMCs, and subpopulation of mesenchymal cells during development 
Nrp2 Coreceptor of Vegfr-3 and semaphorins Transient capillary hypoplasia, defective sprouting (−/−; Yuan et al., 2002; Xu et al., 2010) Venous and lymphatic endothelial cells 
Rac1 Rho GTPase Abnormal migration of LECs from veins (Rac1f/f; Tie1-Cre; D’Amico et al., 2009) Broad 
Clp24 Transmembrane protein Lymphangiectasia, ectopic mural cells (Clp24−/−; Clp24−/−; Vegfr3lacz/+ and Clp24−/−; Vegfr2lacz/+; Saharinen et al., 2010) Endothelial cells 
Tbx1 Transcription factor Hypoplasia and chylous ascites (Tbx1f/f; Tie2-Cre; Chen et al., 2010) Vascular, including LECs 
Ptpn14 (gene trap) Protein tyrosine phosphatase Hyperplasia of dermal lymphatic vessels in 14% of mutants, paw or periorbital edema (Au et al., 2010) Broad, including LECs 
Adrenomedullin signaling 
Adm (adrenomedullinPeptide vasodilator, ligand for Calcrl Hypoplasia of jugular lymph sacs, decreased LEC proliferation, edema (−/−, CalcrlLoxP/–;Tie2-Cre; Fritz-Six et al., 2008) Adrenal medulla, vascular SMCs and endothelial cells, cardiomyocytes 
Ramp2 Coreceptor of Calcrl Hypoplasia of jugular lymph sacs, decreased LEC proliferation, edema (−/−, CalcrlLoxP/–;Tie2-Cre; Fritz-Six et al., 2008) Broad, including LECs 
Calcrl (calcitonin receptor-like) G protein–coupled receptor of adrenomedullin Hypoplasia of jugular lymph sacs, decreased LEC proliferation, edema (−/−, CalcrlLoxP/–;Tie2-Cre; Fritz-Six et al., 2008) Broad, including LECs 
Other 
Vezf Transcription factor Transient jugular lymphatic hypervascularization (+/−; Kuhnert et al., 2005) Broad 
Tie1 Receptor tyrosine kinase Abnormal lymphatic patterning, dilated and disorganized lymphatic vessels (hypomorphic mice on outbred background; D’Amico et al., 2010) Endothelial and hematopoietic cells 
Separation of blood and lymphatic vessels 
Platelet development 
Meis1 Transcription factor Blood-filled lymphatic vessels (−/−; Carramolino et al., 2010) Broad, including developing hematopoietic cells 
Platelet aggregation 
Slp76 Adaptor protein Blood-filled lymphatic vessels, chylous ascites (−/−; Abtahian et al., 2003) Hematopoietic cells 
Plcg2 Phospholipase C, hydrolysis of phospholipids Blood-filled lymphatic vessels, chylous ascites (−/−; Ichise et al., 2009) Broad 
Pdpn (podoplanin) Transmembrane glycoprotein Lymphangiectasia, abnormal lymph transport, lymphedema, blood-filled lymphatic vessels (−/−; Schacht et al., 2003; Bertozzi et al., 2010; Uhrin et al., 2010) LECs, keratinocytes, alveolar type II cells, podocytes 
C1galt1 (encodes T-synthetase) Glycosyltransferase, biosynthesis of core-1–derived O-glycans Blood-filled lymphatic vessels, decreased levels of podoplanin (−/−; Fu et al., 2008) Endothelial and hematopoietic cells 
Clec-2 C-type lectin receptor Blood-filled lymphatic vessels (−/−; Bertozzi et al., 2010; Suzuki-Inoue et al., 2010) Platelets, peripheral blood neutrophils 
Syk Nonreceptor tyrosine kinase Blood-filled lymphatic vessels, chylous ascites, accumulation of myeloid cells in the dermis (−/−, Sykf/f;vav-cre; Abtahian et al., 2003; Bertozzi et al., 2010; Böhmer et al., 2010)  
Other 
Spred1/Spred2 Cytoplasmic adaptor proteins Blood-filled lymphatic vessels (Spred1−/−; Spred2−/−; Taniguchi et al., 2007) Broad, including LECs 
Remodeling, maturation, and valve morphogenesis 
Tie/PI3-kinase signaling 
Akt1 Ser/Thr kinase Capillary hypoplasia, valve agenesis, dilation, and decreased SMC coverage of small collecting lymphatic vessels (−/−; Zhou et al., 2010) Broad 
Angpt2 Growth factor, ligand of Tie receptor tyrosine kinases Hypoplasia, chylous ascites, defective remodeling, and valve agenesis (−/−; Gale et al., 2002; Dellinger et al., 2008) (Lymph) angiogenenic endothelial cells, hematopoietic cells 
Pi3kca (mutation blocking interaction with Ras) Catalytic p110α isoform of PI3-kinase Chylous ascites, hypoplasia, impaired sprouting, and branching of lymphatic capillaries (−/−; Gupta et al., 2007) Broad 
Pik3r1 Regulatory subunits of class IA PI3-kinases Chylous ascites, intestinal lymphangiectasia, impaired sprouting, lymphatic, valve agenesis (−/−; Mouta-Bellum et al., 2009) Broad 
ECM assembly and interactions 
Itga9 Adhesion Chylothorax, lymphatic valve agenesis (−/−; Itga9f/f; Tie2-Cre and Itga9f/f; Cdh5 (PAC)-CreERT2; Bazigou et al., 2009) BECs and LECs (highest in valves), vascular SMCs 
Fn1 (removal of EDA domain) ECM component, ligand of Itg-α9 Failure of lymphatic valve leaflet elongation (−/−; Bazigou et al., 2009) Broad, including lymphatic valves 
Emilin1 Elastic microfibril-associated protein Hyperplasia and abnormal patterning of lymphatic vessels, reduction of anchoring filaments; impaired lymph drainage, increased lymph leakage (−/−; Danussi et al., 2008) Broad, including LECs 
Other 
Aspp1 Cytoplasmic adaptor protein Impaired assembly of lymphatic vessels and collecting lymphatic vessel patterning, accumulation of « lymphatic islands » (−/−; Hirashima et al., 2008) Endothelial cells 
Efnb2 (mutation of PDZ binding site) Ligand of EphB receptor tyrosine kinases Impaired sprouting of capillaries, agenesis of lymphatic valves, ectopic mural cells, chylothorax, retrograde lymph flow (−/−; Mäkinen et al., 2005) Arterial endothelial cells and SMCs, LECs (highest in the valves) 
Foxc2 Transcription factor Impaired patterning of capillaries, no collecting lymphatic vessels, agenesis of lymphatic valves, ectopic mural cells, retrograde lymph flow (−/−; Dagenais et al., 2004; Petrova et al., 2004; Norrmén et al., 2009) Arterial endothelial cells and SMCs, LECs (highest in the valves) 
Elk3 (Net) Transcription factor Lymphangiectasia, chylothorax (−/−; Ayadi et al., 2001) Endothelial cells 
PU.1 Csfr1 Transcription factor receptor for M-CSF1 Hyperplasia and abnormal patterning of dermal lymphatic vessels (−/−; Gordon et al., 2010) Hematopoietic cells (stage dependent) 
Gene symbol Function Lymphatic vascular phenotype Expression pattern 
LEC differentiation 
Prox1 Transcription factor No LECs (−/−), chylous ascites (+/−); loss of LEC identity, chylous ascites, obesity (Prox1f/f; Tie2-Cre; Wigle et al., 1999; Harvey et al., 2005; Johnson et al., 2008) LECs, hepatocytes, lens fiber cells, pancreatic, lung, and intestinal endocrine cells, skeletal muscle, cardiomyocytes 
Sox18 Transcription factor No LECs(−/−, on C57BL/6J background); abnormal patterning (+/−); edema and chylous ascites (heterozygous ragged mutants; Pennisi et al., 2000; François et al., 2008) Endothelial cells 
Coup-TFII (NR2F2Transcription factor No LECs (−/−, deletion at or before E9.5); edema, loss of LEC identity, and sprouting (−/−, deletion at later stages; Lin et al., 2010; Srinivasan et al., 2010) Endothelial cells and SMCs 
Lymphangiogenesis 
Vegf-c–Vegfr-3 pathway 
Vegfr3 Receptor tyrosine kinase Hypoplasia, chylous ascites (+/Chy, ENU-induced mutation, loss of tyrosine kinase activity; Karkkainen et al., 2001); lymph sacs formed and defective sprouting (homozygous deletion of ligand-binding domain; Zhang et al., 2010) LECs, fenestrated blood vascular endothelial cells, blood vascular endothelial cells in tumors and during early embryogenesis 
Vegfc Growth factor, ligand for Vegfr-3 No sprouting of LECs from veins (−/−); hypoplasia, chylous ascites (+/−; Karkkainen et al., 2003) Macrophages, SMCs, and subpopulation of mesenchymal cells during development 
Nrp2 Coreceptor of Vegfr-3 and semaphorins Transient capillary hypoplasia, defective sprouting (−/−; Yuan et al., 2002; Xu et al., 2010) Venous and lymphatic endothelial cells 
Rac1 Rho GTPase Abnormal migration of LECs from veins (Rac1f/f; Tie1-Cre; D’Amico et al., 2009) Broad 
Clp24 Transmembrane protein Lymphangiectasia, ectopic mural cells (Clp24−/−; Clp24−/−; Vegfr3lacz/+ and Clp24−/−; Vegfr2lacz/+; Saharinen et al., 2010) Endothelial cells 
Tbx1 Transcription factor Hypoplasia and chylous ascites (Tbx1f/f; Tie2-Cre; Chen et al., 2010) Vascular, including LECs 
Ptpn14 (gene trap) Protein tyrosine phosphatase Hyperplasia of dermal lymphatic vessels in 14% of mutants, paw or periorbital edema (Au et al., 2010) Broad, including LECs 
Adrenomedullin signaling 
Adm (adrenomedullinPeptide vasodilator, ligand for Calcrl Hypoplasia of jugular lymph sacs, decreased LEC proliferation, edema (−/−, CalcrlLoxP/–;Tie2-Cre; Fritz-Six et al., 2008) Adrenal medulla, vascular SMCs and endothelial cells, cardiomyocytes 
Ramp2 Coreceptor of Calcrl Hypoplasia of jugular lymph sacs, decreased LEC proliferation, edema (−/−, CalcrlLoxP/–;Tie2-Cre; Fritz-Six et al., 2008) Broad, including LECs 
Calcrl (calcitonin receptor-like) G protein–coupled receptor of adrenomedullin Hypoplasia of jugular lymph sacs, decreased LEC proliferation, edema (−/−, CalcrlLoxP/–;Tie2-Cre; Fritz-Six et al., 2008) Broad, including LECs 
Other 
Vezf Transcription factor Transient jugular lymphatic hypervascularization (+/−; Kuhnert et al., 2005) Broad 
Tie1 Receptor tyrosine kinase Abnormal lymphatic patterning, dilated and disorganized lymphatic vessels (hypomorphic mice on outbred background; D’Amico et al., 2010) Endothelial and hematopoietic cells 
Separation of blood and lymphatic vessels 
Platelet development 
Meis1 Transcription factor Blood-filled lymphatic vessels (−/−; Carramolino et al., 2010) Broad, including developing hematopoietic cells 
Platelet aggregation 
Slp76 Adaptor protein Blood-filled lymphatic vessels, chylous ascites (−/−; Abtahian et al., 2003) Hematopoietic cells 
Plcg2 Phospholipase C, hydrolysis of phospholipids Blood-filled lymphatic vessels, chylous ascites (−/−; Ichise et al., 2009) Broad 
Pdpn (podoplanin) Transmembrane glycoprotein Lymphangiectasia, abnormal lymph transport, lymphedema, blood-filled lymphatic vessels (−/−; Schacht et al., 2003; Bertozzi et al., 2010; Uhrin et al., 2010) LECs, keratinocytes, alveolar type II cells, podocytes 
C1galt1 (encodes T-synthetase) Glycosyltransferase, biosynthesis of core-1–derived O-glycans Blood-filled lymphatic vessels, decreased levels of podoplanin (−/−; Fu et al., 2008) Endothelial and hematopoietic cells 
Clec-2 C-type lectin receptor Blood-filled lymphatic vessels (−/−; Bertozzi et al., 2010; Suzuki-Inoue et al., 2010) Platelets, peripheral blood neutrophils 
Syk Nonreceptor tyrosine kinase Blood-filled lymphatic vessels, chylous ascites, accumulation of myeloid cells in the dermis (−/−, Sykf/f;vav-cre; Abtahian et al., 2003; Bertozzi et al., 2010; Böhmer et al., 2010)  
Other 
Spred1/Spred2 Cytoplasmic adaptor proteins Blood-filled lymphatic vessels (Spred1−/−; Spred2−/−; Taniguchi et al., 2007) Broad, including LECs 
Remodeling, maturation, and valve morphogenesis 
Tie/PI3-kinase signaling 
Akt1 Ser/Thr kinase Capillary hypoplasia, valve agenesis, dilation, and decreased SMC coverage of small collecting lymphatic vessels (−/−; Zhou et al., 2010) Broad 
Angpt2 Growth factor, ligand of Tie receptor tyrosine kinases Hypoplasia, chylous ascites, defective remodeling, and valve agenesis (−/−; Gale et al., 2002; Dellinger et al., 2008) (Lymph) angiogenenic endothelial cells, hematopoietic cells 
Pi3kca (mutation blocking interaction with Ras) Catalytic p110α isoform of PI3-kinase Chylous ascites, hypoplasia, impaired sprouting, and branching of lymphatic capillaries (−/−; Gupta et al., 2007) Broad 
Pik3r1 Regulatory subunits of class IA PI3-kinases Chylous ascites, intestinal lymphangiectasia, impaired sprouting, lymphatic, valve agenesis (−/−; Mouta-Bellum et al., 2009) Broad 
ECM assembly and interactions 
Itga9 Adhesion Chylothorax, lymphatic valve agenesis (−/−; Itga9f/f; Tie2-Cre and Itga9f/f; Cdh5 (PAC)-CreERT2; Bazigou et al., 2009) BECs and LECs (highest in valves), vascular SMCs 
Fn1 (removal of EDA domain) ECM component, ligand of Itg-α9 Failure of lymphatic valve leaflet elongation (−/−; Bazigou et al., 2009) Broad, including lymphatic valves 
Emilin1 Elastic microfibril-associated protein Hyperplasia and abnormal patterning of lymphatic vessels, reduction of anchoring filaments; impaired lymph drainage, increased lymph leakage (−/−; Danussi et al., 2008) Broad, including LECs 
Other 
Aspp1 Cytoplasmic adaptor protein Impaired assembly of lymphatic vessels and collecting lymphatic vessel patterning, accumulation of « lymphatic islands » (−/−; Hirashima et al., 2008) Endothelial cells 
Efnb2 (mutation of PDZ binding site) Ligand of EphB receptor tyrosine kinases Impaired sprouting of capillaries, agenesis of lymphatic valves, ectopic mural cells, chylothorax, retrograde lymph flow (−/−; Mäkinen et al., 2005) Arterial endothelial cells and SMCs, LECs (highest in the valves) 
Foxc2 Transcription factor Impaired patterning of capillaries, no collecting lymphatic vessels, agenesis of lymphatic valves, ectopic mural cells, retrograde lymph flow (−/−; Dagenais et al., 2004; Petrova et al., 2004; Norrmén et al., 2009) Arterial endothelial cells and SMCs, LECs (highest in the valves) 
Elk3 (Net) Transcription factor Lymphangiectasia, chylothorax (−/−; Ayadi et al., 2001) Endothelial cells 
PU.1 Csfr1 Transcription factor receptor for M-CSF1 Hyperplasia and abnormal patterning of dermal lymphatic vessels (−/−; Gordon et al., 2010) Hematopoietic cells (stage dependent) 

ENU, N-ethyl-N-nitrosourea; PAC, P1-derived artificial chromosome.

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