LMX1B

UniProt ID: O60663
Organism: Homo sapiens
Review Status: COMPLETE
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Gene Description

LMX1B is a nuclear LIM-homeodomain DNA-binding transcriptional activator that recognizes regulatory DNA and works with context-dependent transcriptional coregulators. It establishes and maintains tissue-selective gene-expression programs in podocytes, the dorsal limb, and neuronal lineages including midbrain dopaminergic and central serotonergic neurons. In human cells, LMX1B directly promotes macroautophagy-gene expression and autophagic flux and binds ATG8-family ubiquitin-like proteins; this nutrient- and compartment-sensitive interaction enhances its transcriptional output and cellular stress resilience. Pathogenic LMX1B variants cause nail-patella syndrome and kidney-limited glomerular disease.

Existing Annotations Review

GO Term Evidence Action Reason
GO:0005634 nucleus
IBA
GO_REF:0000033
ACCEPT
Summary: Nuclear localization is a core feature of this LIM-homeodomain transcription factor and is independently supported by direct imaging of human dopaminergic neurons.
Reason: The IBA is biologically coherent with LMX1B's sequence-specific nuclear transcriptional activity. Human differentiated dopaminergic neurons show LMX1B in nuclei, so there is no indication of a faulty phylogenetic transfer.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
FB:FBgn0267978
MGI:MGI:1096343
MGI:MGI:1100513
MGI:MGI:1306803
MGI:MGI:96785
MGI:MGI:99783
PANTHER:PTN000654232
RGD:71074
UniProtKB:O60663
UniProtKB:O97581
UniProtKB:P29674
UniProtKB:P53411
UniProtKB:Q9UPM6
WB:WBGene00000438
WB:WBGene00002987
WB:WBGene00002988
WB:WBGene00003167
WB:WBGene00006654
Supporting Evidence:
PMID:24399192
LMX1b was expressed in all nuclei (inset). There was also unspecific cytoplasmic fluorescence.
GO:0006357 regulation of transcription by RNA polymerase II
IBA
GO_REF:0000033
ACCEPT
Summary: This IBA correctly captures the broad, unsigned process by which LMX1B regulates RNA polymerase II transcription across target and cofactor contexts.
Reason: GO:0006357 is the valid RNA polymerase II regulation parent of the directly established positive-regulation processes and accurately preserves the broad phylogenetic inference. More specific positive-regulation annotations are retained separately. LDB1 can attenuate the magnitude of LMX1B-mediated activation, but this is not interpreted as evidence for negative regulation.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
FB:FBgn0267978
MGI:MGI:102673
MGI:MGI:1100513
MGI:MGI:1888519
MGI:MGI:96785
PANTHER:PTN004680160
UniProtKB:C4TJC6
UniProtKB:P29674
UniProtKB:Q969G2
UniProtKB:Q9UBR4
WB:WBGene00000438
WB:WBGene00002987
WB:WBGene00002988
WB:WBGene00003167
WB:WBGene00006654
Supporting Evidence:
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
PMID:10767331
While co--transfections of E47/shPan1 with LMX1B result in a synergistic effect on reporter activity, LDB1 down-regulated LMX1B-mediated transactivation irrespective of E47/shPan1.
GO:0030182 neuron differentiation
IBA
GO_REF:0000033
KEEP AS NON CORE
Summary: The broad neuron-differentiation inference is biologically defensible, but it describes one developmental context of a pleiotropic transcription factor rather than its molecular core.
Reason: LMX1B contributes to development and maintenance of several neuronal populations, including mesodiencephalic dopaminergic neurons. Retain the broad process as contextual because LMX1B also has independent renal, limb, ocular, and serotonergic developmental roles.
Propagation Review
Root cause: NO FAILURE NON CORE
Sources checked:
FB:FBgn0002023
FB:FBgn0267978
MGI:MGI:101776
MGI:MGI:102673
MGI:MGI:107792
MGI:MGI:1096343
MGI:MGI:1100513
MGI:MGI:1306803
MGI:MGI:1888519
MGI:MGI:96785
MGI:MGI:99783
PANTHER:PTN000654232
RGD:71076
UniProtKB:P53411
UniProtKB:Q8UVR3
UniProtKB:Q90881
WB:WBGene00000438
WB:WBGene00002987
WB:WBGene00002988
WB:WBGene00003000
WB:WBGene00003167
WB:WBGene00006654
ZFIN:ZDB-GENE-050114-2
ZFIN:ZDB-GENE-050114-3
ZFIN:ZDB-GENE-050417-210
ZFIN:ZDB-GENE-051220-1
ZFIN:ZDB-GENE-060728-1
ZFIN:ZDB-GENE-980526-131
Supporting Evidence:
PMID:12897786
A major determinant in the cascades is an LIM homeodomain-containing gene, Lmx1b, which is required for the development of all 5-HT neurons in the central nervous system.
PMID:23308148
The LIM homeodomain transcription factor Lmx1b is essential for the development of the isthmic organizer and mesodiencephalic dopaminergic neurons.
GO:0000977 RNA polymerase II transcription regulatory region sequence-specific DNA binding
IBA
GO_REF:0000033
ACCEPT
Summary: Sequence-specific binding to RNA polymerase II regulatory DNA is a core molecular function of LMX1B's homeodomain.
Reason: Direct binding of LMX1B to regulatory sequences and activation through those sequences supports the IBA at an appropriate level of specificity.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
FB:FBgn0267978
MGI:MGI:1100513
MGI:MGI:1888519
MGI:MGI:96785
PANTHER:PTN004680160
WB:WBGene00000438
WB:WBGene00002987
WB:WBGene00006654
Supporting Evidence:
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
GO:0000981 DNA-binding transcription factor activity, RNA polymerase II-specific
IBA
GO_REF:0000033
ACCEPT
Summary: RNA polymerase II-specific DNA-binding transcription factor activity is the central molecular function of LMX1B.
Reason: The homeodomain supplies selective regulatory-DNA binding and LMX1B activates mammalian target-gene reporters; the IBA therefore captures the correct core activity without overclaiming a particular target or tissue.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
MGI:MGI:1100513
MGI:MGI:1888519
MGI:MGI:96785
PANTHER:PTN004680160
UniProtKB:Q969G2
UniProtKB:Q9UBR4
WB:WBGene00002987
WB:WBGene00003167
WB:WBGene00006654
Supporting Evidence:
PMID:11956244
Thus, LMX1B regulates the expression of multiple podocyte genes critical for podocyte differentiation and function.
GO:0000981 DNA-binding transcription factor activity, RNA polymerase II-specific
IEA
GO_REF:0000002
ACCEPT
Summary: The InterPro homeobox-signature mapping correctly predicts LMX1B's RNA polymerase II-specific DNA-binding transcription factor activity.
Reason: The prediction is independently corroborated by direct LMX1B regulatory-DNA binding and transcriptional activation. The mapping is redundant with stronger evidence but not biologically erroneous.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
InterPro:IPR017970
Supporting Evidence:
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
GO:0003677 DNA binding
IEA
GO_REF:0000002
MODIFY
Summary: The homeodomain mapping is directionally correct, but generic DNA binding loses the experimentally established sequence specificity and double-stranded substrate.
Reason: LMX1B recognizes specific regulatory DNA sequences through its homeodomain. Replace the broad parent with sequence-specific double-stranded DNA binding, which is also directly supported in the existing annotation set.
Propagation Review
Root cause: TERM SCOPING PROBLEM
Failure modes: GRANULARITY MISMATCH
Sources checked:
InterPro:IPR001356
Supporting Evidence:
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
GO:0005634 nucleus
IEA
GO_REF:0000044
ACCEPT
Summary: The UniProt subcellular-location mapping correctly places LMX1B in the nucleus.
Reason: Nuclear localization is independently demonstrated in human dopaminergic neurons and is the expected site of LMX1B's core transcription-factor activity.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
UniProtKB-SubCell:SL-0191
Supporting Evidence:
PMID:24399192
LMX1b was expressed in all nuclei (inset). There was also unspecific cytoplasmic fluorescence.
GO:0006355 regulation of DNA-templated transcription
IEA
GO_REF:0000002
MODIFY
Summary: The InterPro mapping identifies a true unsigned transcriptional role, but its DNA-templated-transcription parent does not specify the relevant RNA polymerase.
Reason: Refine the generic process to regulation of transcription by RNA polymerase II, the appropriate broad parent supported by the InterPro transcription-factor mapping. Direct experiments support separate positive-regulation rows, while this electronic mapping does not itself establish a signed regulatory effect.
Propagation Review
Root cause: TERM SCOPING PROBLEM
Failure modes: GRANULARITY MISMATCH
Sources checked:
InterPro:IPR017970
Supporting Evidence:
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
GO:0043565 sequence-specific DNA binding
IEA
GO_REF:0000117
MODIFY
Summary: Sequence-specific DNA binding is correct, but LMX1B's regulatory substrate is double-stranded DNA, making the existing term less precise than a supported child term.
Reason: The ARBA prediction captures specificity but not DNA strandedness. Direct LMX1B binding-site assays and the existing IDA annotation support sequence-specific double-stranded DNA binding.
Propagation Review
Root cause: TERM SCOPING PROBLEM
Failure modes: GRANULARITY MISMATCH
Sources checked:
ARBA:ARBA00026779
Supporting Evidence:
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
GO:0005515 protein binding
IPI
PMID:12792813
Confirmation of CLIM2/LMX1B interaction by yeast two-hybrid ...
MODIFY
Summary: The LMX1B-LDB1 interaction is supported, but bare protein binding is uninformative; from LMX1B's perspective this is binding to a transcriptional coregulator.
Reason: LDB1/CLIM2 is a LIM-homeodomain transcriptional coregulator. GO:0001221 therefore describes what LMX1B binds. LIM-domain binding would describe the reciprocal activity of LDB1 binding LMX1B's LIM domains, not LMX1B's activity, and is inappropriate for this gene-product annotation.
Supporting Evidence:
PMID:12792813
By the yeast two-hybrid screening we detected the CLIM2 gene as a LMX1B interactor, confirming previous reports which described the same interaction by biochemical methods.
GO:0005515 protein binding
IPI
PMID:20211142
An atlas of combinatorial transcriptional regulation in mous...
MODIFY
Summary: The interaction atlas supports LMX1B association with LDB1 and SSBP3, but the generic protein-binding label obscures their role as LIM-homeodomain transcriptional coregulators.
Reason: Replace generic protein binding with transcription coregulator binding. LDB1 is the established LIM-domain-interacting coregulator and SSBP3 is a component of LDB1-centered transcriptional complexes. The replacement is assigned from LMX1B's perspective; LIM-domain binding would reverse the direction of the molecular function.
Supporting Evidence:
PMID:10767331
Transfection studies showed that both the LIM domain-interacting protein, LDB1, and the helix-loop-helix protein, E47/shPan1, can regulate LMX1B action.
GO:0005515 protein binding
IPI
PMID:30833792
A protein-interaction network of interferon-stimulated genes...
MODIFY
Summary: This high-throughput IPI records the reproducible LMX1B-SSBP3 interaction, but bare protein binding is not functionally informative.
Reason: SSBP3 functions as a transcriptional coregulator in LDB1/LIM-homeodomain complexes, making transcription coregulator binding the appropriate gene-product-perspective replacement. The immune-network assay context does not itself make an innate-immune role for LMX1B.
GO:0005515 protein binding
IPI
PMID:33961781
Dual proteome-scale networks reveal cell-specific remodeling...
MODIFY
Summary: Proteome-scale affinity purification recovers LDB1 and SSBP3 with LMX1B, but generic protein binding should be replaced by the shared mechanistic class of these partners.
Reason: Both partners participate in LIM-homeodomain transcriptional regulatory complexes, so transcription coregulator binding is informative and directionally correct for LMX1B. The proteome-scale cell-line setting supports physical association but does not establish a tissue-specific biological process.
Supporting Evidence:
PMID:33961781
Through affinity-purification mass spectrometry, we have created two proteome-scale, cell-line-specific interaction networks.
GO:0005515 protein binding
IPI
PMID:40205054
Multimodal cell maps as a foundation for structural and func...
MODIFY
Summary: The multimodal interaction map again supports LMX1B association with LDB1 and SSBP3, while generic protein binding fails to capture their transcriptional role.
Reason: Replace with transcription coregulator binding. LDB1 and SSBP3 are partners in transcriptional regulatory complexes, and this term describes LMX1B binding those partners without incorrectly assigning the reciprocal LIM-domain-binding activity to LMX1B.
GO:0000977 RNA polymerase II transcription regulatory region sequence-specific DNA binding
ISS
GO_REF:0000024
ACCEPT
Summary: Manual transfer from mouse Lmx1b correctly captures the conserved, core regulatory-DNA-binding activity of human LMX1B.
Reason: The mouse ortholog is one-to-one and the transfer is independently corroborated by human and mammalian binding-site assays. No paralog-specific divergence is evident for the homeodomain activity.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
UniProtKB:O88609
Supporting Evidence:
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
GO:0000981 DNA-binding transcription factor activity, RNA polymerase II-specific
ISS
GO_REF:0000024
ACCEPT
Summary: The RNA polymerase II-specific transcription-factor activity is core; the five extensions preserve the target scope of the manually transferred mouse annotation.
Reason: Retain the manually curated orthology transfer, because mammalian evidence supports LMX1B regulation of this midbrain network and human assays independently establish the core molecular activity. Evidence strength is not identical for every WNT1, PITX3, NR4A2, LMX1A, and MSX1 target relationship in human cells, but that caveat does not invalidate the conserved transcription-factor activity.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
UniProtKB:O88609
Supporting Evidence:
PMID:23308148
The (partial) loss of Pitx3 and later of Th in the Lmx1b null mutant suggest that Lmx1b may act as an upstream activator of these genes in the development of mdDA neurons [1]–[3], or Lmx1b may be involved in specifying the dopaminergic niche in the midbrain region.
GO:1990837 sequence-specific double-stranded DNA binding
IDA
PMID:28473536
Impact of cytosine methylation on DNA binding specificities ...
ACCEPT
Summary: Methylation-sensitive SELEX directly supports sequence-specific double-stranded DNA binding by human LMX1B; this is an appropriate core molecular-function term.
Reason: The high-throughput study experimentally profiled human transcription-factor DNA specificity. Independent target-regulatory-region assays corroborate the sequence-specific double-stranded DNA-binding function without requiring a more motif-specific GO term.
Supporting Evidence:
PMID:28473536
By analysis of 542 human TFs with methylation-sensitive SELEX (systematic evolution of ligands by exponential enrichment), we found that there are also many TFs that prefer CpG-methylated sequences.
PMID:11956244
We identified several LMX1B binding sites in the putative regulatory regions of both CD2AP and NPHS2 (podocin) and demonstrated that LMX1B binds to these sequences in vitro and can activate transcription through them in cotransfection assays.
GO:0000785 chromatin
ISA
GO_REF:0000113
ACCEPT
Summary: Chromatin is an appropriate active location for a sequence-specific DNA-binding transcription factor and is corroborated by direct LMX1B promoter occupancy assays.
Reason: TFClass correctly classifies LMX1B as a LIM-homeodomain DNA-binding transcription factor. Direct chromatin immunoprecipitation in human cells confirms occupancy at target promoters, supporting chromatin as a core activity location.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
tfclass:3.1.5
Supporting Evidence:
PMID:37014324
Using an arbitrary twofold cut-off for targets of interest, promoter occupancy was confirmed for several core autophagy genes, including ULK1, ATG3, ATG16L1, UVRAG, as well as the autolysosomal transcription factor TFEB and the receptors and/or mitophagy genes, NDP52, OPTN, and PINK1 (Fig. 1 A).
GO:0000981 DNA-binding transcription factor activity, RNA polymerase II-specific
ISA
GO_REF:0000113
ACCEPT
Summary: TFClass correctly assigns RNA polymerase II-specific DNA-binding transcription factor activity to LMX1B.
Reason: The classification is consistent with the homeodomain and with direct regulatory-DNA binding, reporter activation, and endogenous target-gene regulation. It captures the core molecular function at a suitable level of specificity.
Propagation Review
Root cause: NO FAILURE CORE
Sources checked:
tfclass:3.1.5
Supporting Evidence:
PMID:11956244
Thus, LMX1B regulates the expression of multiple podocyte genes critical for podocyte differentiation and function.
GO:0045944 positive regulation of transcription by RNA polymerase II
TAS
PMID:24431302
Wnt signaling in midbrain dopaminergic neuron development an...
KEEP AS NON CORE
Summary: LMX1B-dependent activation of WNT1 transcription at the midbrain-hindbrain boundary is biologically coherent, but this extended annotation is a specific developmental context rather than the gene's general core function.
Reason: Retain the curator-authored TAS annotation. The cited review is not direct primary evidence, but full-text mouse work independently places Lmx1b upstream of Wnt1 in isthmic-organizer development. The tissue and dopaminergic-differentiation extensions make the row appropriately contextual.
Supporting Evidence:
PMID:23308148
This possibility is underlined by the fact that Lmx1b is also involved in regulation of Fgf8 and Wnt1, and several isthmus-related transcription factors, and it is essential for inductive activity of the isthmic organizer (IsO) [4], [5].
GO:0045944 positive regulation of transcription by RNA polymerase II
ISS
GO_REF:0000024
KEEP AS NON CORE
Summary: Positive transcriptional regulation of the transferred midbrain target set is supported in mouse, but the target-specific extensions describe a developmental program rather than the general molecular core.
Reason: Manual orthology transfer from mouse Lmx1b is reasonable and is supported by conserved human transcription-factor activity. Retain as non-core because the WNT1, PITX3, NR4A2, and LMX1A target restrictions are developmental-context statements and are not all equally demonstrated as direct targets in human cells.
Propagation Review
Root cause: NO FAILURE NON CORE
Sources checked:
UniProtKB:O88609
Supporting Evidence:
PMID:23308148
The (partial) loss of Pitx3 and later of Th in the Lmx1b null mutant suggest that Lmx1b may act as an upstream activator of these genes in the development of mdDA neurons [1]–[3], or Lmx1b may be involved in specifying the dopaminergic niche in the midbrain region.
GO:0071542 dopaminergic neuron differentiation
ISS
GO_REF:0000024
KEEP AS NON CORE
Summary: Mouse Lmx1b evidence supports a role in dopaminergic neuron development, but this is one tissue-specific developmental role of a pleiotropic transcription factor.
Reason: Retain the manually transferred process as contextual. Lmx1b is important for establishment of the isthmic organizer and the mesodiencephalic dopaminergic field, although stage- and cell-type-specific knockout studies make its role more nuanced than an invariant cell-autonomous differentiation requirement.
Propagation Review
Root cause: NO FAILURE NON CORE
Sources checked:
UniProtKB:O88609
Supporting Evidence:
PMID:23308148
The LIM homeodomain transcription factor Lmx1b is essential for the development of the isthmic organizer and mesodiencephalic dopaminergic neurons.
GO:0005634 nucleus
IDA
PMID:24399192
Differentiation of human epidermal neural crest stem cells (...
ACCEPT
Summary: Direct immunocytochemistry places human LMX1B in nuclei of differentiated dopaminergic neurons.
Reason: The figure description explicitly reports nuclear LMX1B staining in all scored day-25 dopaminergic neurons. The cell-type extension accurately preserves the experimental context while nucleus remains a core activity location.
Supporting Evidence:
PMID:24399192
LMX1b was expressed in all nuclei (inset). There was also unspecific cytoplasmic fluorescence.
GO:0006355 regulation of DNA-templated transcription
IDA
PMID:10767331
LMX1B transactivation and expression in nail-patella syndrom...
MODIFY
Summary: The experiment demonstrates transcriptional activation, but regulation of DNA-templated transcription is an unsigned and polymerase-unspecified parent.
Reason: LMX1B activates reporter transcription, and independent target-site studies show positive transcriptional regulation. Replace the generic process with positive regulation of transcription by RNA polymerase II.
Supporting Evidence:
PMID:10767331
Mutant LMX1B proteins containing human mutations affecting each of the helices or the N-terminal arm of the homeodomain abolished transactivation, while LIM B and truncation mutations retained residual activity.
GO:0009953 dorsal/ventral pattern formation
ISS
GO_REF:0000024
KEEP AS NON CORE
Summary: The mouse-ortholog transfer correctly captures LMX1B's conserved role in dorsal-ventral patterning, especially dorsal limb fate, but this is a developmental context rather than its molecular core.
Reason: Human nail-patella phenotypes and mouse loss-of-function data strongly support the process. Retain as non-core because the term describes a pleiotropic developmental outcome downstream of LMX1B's transcription-factor activity.
Propagation Review
Root cause: NO FAILURE NON CORE
Sources checked:
UniProtKB:O88609
Supporting Evidence:
PMID:10767331
Lmx1b, a member of the LIM homeodomain protein family, is essential for the specification of dorsal limb fates at the zeugopodal and autopodal level in vertebrates.
GO:0003700 DNA-binding transcription factor activity
IDA
PMID:10767331
LMX1B transactivation and expression in nail-patella syndrom...
MODIFY
Summary: The direct reporter assay establishes DNA-binding transcription-factor activity, but the generic term omits both the activating sign and RNA polymerase II context.
Reason: Replace with DNA-binding transcription activator activity, RNA polymerase II-specific. LMX1B's homeodomain binds regulatory DNA and the tested wild-type protein positively drives reporter and endogenous target-gene transcription.
Supporting Evidence:
PMID:10767331
Mutant LMX1B proteins containing human mutations affecting each of the helices or the N-terminal arm of the homeodomain abolished transactivation, while LIM B and truncation mutations retained residual activity.
GO:0005634 nucleus
IDA
PMID:10767331
LMX1B transactivation and expression in nail-patella syndrom...
ACCEPT
Summary: Nuclear localization is correct for LMX1B and is corroborated by later direct human-cell imaging.
Reason: The source paper is abstract-only in the local cache, so its precise localization assay cannot be re-audited; the experimental annotation should not be overruled. Independent full-text imaging shows human LMX1B in dopaminergic-neuron nuclei, and nuclear localization is consistent with its core transcription-factor function.
Supporting Evidence:
PMID:24399192
LMX1b was expressed in all nuclei (inset). There was also unspecific cytoplasmic fluorescence.
GO:0030182 neuron differentiation
ISS
GO_REF:0000024
KEEP AS NON CORE
Summary: Broad neuron differentiation is a defensible transfer from mouse Lmx1b, but it is less specific than the dopaminergic context and represents a secondary developmental role.
Reason: Retain the curator-judged orthology transfer. LMX1B contributes to development and maintenance of neuronal populations, but its molecular core is sequence-specific transcriptional regulation and it also has substantial non-neuronal functions.
Propagation Review
Root cause: NO FAILURE NON CORE
Sources checked:
UniProtKB:O88609
Supporting Evidence:
PMID:12897786
A major determinant in the cascades is an LIM homeodomain-containing gene, Lmx1b, which is required for the development of all 5-HT neurons in the central nervous system.
PMID:37014324
The LIM homeodomain transcription factors LMX1A and LMX1B are essential mediators of midbrain dopaminergic neuronal (mDAN) differentiation and survival.
GO:0016239 positive regulation of macroautophagy
IMP
PMID:37014324
ATG8-dependent LMX1B-autophagy crosstalk shapes human midbra...
NEW
Summary: Direct perturbation and rescue experiments in human cells show that LMX1B promotes basal and nutrient-stress-induced macroautophagic flux.
Reason: LMX1B depletion or knockout reduced expression of multiple autophagy genes and dampened autophagic flux in HEK293T cells, while LMX1B suppression in human iPSC-derived midbrain dopaminergic neurons reduced autophagy-gene expression and autophagosome assembly sites. These direct human phenotypes support positive regulation of macroautophagy without relying on mouse orthology.
Supporting Evidence:
PMID:37014324
Together, these data demonstrate that LMX1B contributes to the control of autophagy gene expression in HEK293T cells, and that LMX1B depletion dampens basal/housekeeping and nutrient stress-induced autophagic flux responses.
GO:0032182 ubiquitin-like protein binding
IPI
PMID:37014324
ATG8-dependent LMX1B-autophagy crosstalk shapes human midbra...
NEW
Summary: Endogenous co-immunoprecipitation in human HEK293T cells demonstrates binding between native LMX1B and the ATG8-family ubiquitin-like protein LC3B.
Reason: The endogenous LMX1B-LC3B co-immunoprecipitation supports ubiquitin-like protein binding and is encoded with LC3B as the sole supporting entity. Complementary tagged assays recovered LMX1B with all six human ATG8-family proteins, but those five additional partners are not added as entities because their evidence was not an endogenous interaction assay. This is a regulated cofactor interaction, not evidence that LMX1B is a stable complex subunit.
Supporting Evidence:
PMID:37014324
Importantly, we observed coIP of endogenous LC3B using antibodies against native LMX1B in HEK293T cells (Fig. 5 E).
GO:0072248 metanephric podocyte differentiation
ISO
PMID:11956244
Transcriptional induction of slit diaphragm genes by Lmx1b i...
NEW
Summary: Mouse Lmx1b loss causes developmental arrest and structural dysplasia of podocytes in the developing definitive kidney, supporting transfer of metanephric podocyte differentiation to human LMX1B.
Reason: The mouse null phenotype directly establishes a requirement for Lmx1b in metanephric podocyte differentiation, and the one-to-one human ortholog has a conserved renal disease role. The more specific metanephric child term matches the mammalian definitive-kidney experiment. The ISO annotation does not assert that any proposed COL4A3, COL4A4, NPHS2, or CD2AP target relationship is universal in human podocytes.
Propagation Review
Root cause: NO FAILURE NON CORE
Sources checked:
UniProtKB:O88609 SUPPORTS TRANSFER
The experimentally tested mouse Lmx1b ortholog supplies the podocyte differentiation phenotype; the proposed human annotation is an orthology transfer.
Supporting Evidence:
PMID:11956244
Here, we show that Lmx1b(-/-) podocytes have reduced numbers of foot processes, are dysplastic, and lack typical slit diaphragms, indicating an arrest in development.
GO:0032956 regulation of actin cytoskeleton organization
ISO
PMID:23990680
LMX1B is essential for the maintenance of differentiated pod...
NEW
Summary: Conditional loss of mouse Lmx1b in adult podocytes dysregulates actin cytoskeleton organization, supporting a podocyte-restricted transfer to human LMX1B.
Reason: The acute adult mouse phenotype supports regulation of actin cytoskeleton organization as part of LMX1B-dependent podocyte maintenance. Human-podocyte ChIP and gel-shift assays independently show direct promoter recognition of ABRA and ARL4C, genes identified in the same study as actin-cytoskeleton-associated targets. Thus, the regulation is transcription-mediated rather than inferred from a bare downstream phenotype. ISO and the podocyte extension preserve the species and cell-type boundaries without asserting that this target program is universal.
Propagation Review
Root cause: NO FAILURE NON CORE
Sources checked:
UniProtKB:O88609 SUPPORTS TRANSFER
Conditional mouse Lmx1b inactivation supplies the adult podocyte phenotype; the proposed human annotation is an orthology transfer.
Supporting Evidence:
PMID:23990680
Cell biological and biophysical experiments with primary podocytes isolated after 1 week of Lmx1b inactivation indicated dysregulation of actin cytoskeleton organization
PMID:23990680
Chromatin immunoprecipitation experiments in conditionally immortalized human podocytes and gel shift assays showed that LMX1B recognizes AT-rich binding sites (FLAT elements) in the promoter regions of ABRA and ARL4C

Core Functions

LMX1B binds sequence-specific regulatory DNA through its homeodomain and acts as an RNA polymerase II transcriptional activator. In human cells this activity directly drives an autophagy-gene program and promotes macroautophagic flux. ATG8-family binding is a context-dependent regulatory input that enhances this transcriptional output, rather than a separate conserved core function. LMX1B's podocyte, dorsal-limb, and neuronal developmental programs are supported principally by mouse loss-of-function and orthology evidence and are not treated as universal direct human target-gene relationships.

Supporting Evidence:
  • PMID:10767331
    Mutant LMX1B proteins containing human mutations affecting each of the helices or the N-terminal arm of the homeodomain abolished transactivation, while LIM B and truncation mutations retained residual activity.
  • PMID:37014324
    Using an arbitrary twofold cut-off for targets of interest, promoter occupancy was confirmed for several core autophagy genes, including ULK1, ATG3, ATG16L1, UVRAG, as well as the autolysosomal transcription factor TFEB and the receptors and/or mitophagy genes, NDP52, OPTN, and PINK1 (Fig. 1 A).
  • PMID:37014324
    Together, these data demonstrate that LMX1B contributes to the control of autophagy gene expression in HEK293T cells, and that LMX1B depletion dampens basal/housekeeping and nutrient stress-induced autophagic flux responses.
  • PMID:37014324
    Ξ”308-317 LMX1B bound poorly to all ATG8s, failed to rescue autophagy gene expression, was unable to stimulate FLAT-promoter luciferase reporter expression, and crucially, failed to protect against rotenone in HEK293T cells and iPSC-derived human mDANs.

References

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Suggested Questions for Experts

Q: Which endogenous direct LMX1B targets, rather than secondary differentiation markers, are required for human podocyte maturation and long-term filtration-barrier maintenance?

Suggested experts: podocyte developmental biologists, human glomerular-disease geneticists

Q: Which endogenous LC3/GABARAP paralogs modulate LMX1B transcription in human midbrain dopaminergic neurons, and how does nutrient state redistribute the functional interaction between nucleus and cytosol?

Suggested experts: autophagy interaction specialists, human midbrain dopaminergic-neuron biologists

Q: Do O60663-1, O60663-2, and O60663-3 have distinct transcriptional targets, interaction partners, or tissue-specific functions in vivo?

Suggested experts: LMX1B isoform specialists

Suggested Experiments

Experiment: Engineer an endogenous degron in human iPSC-derived podocytes and perform a short time course of LMX1B CUT&RUN, PRO-seq, single-cell RNA-seq, quantitative cytoskeletal imaging, and filtration-barrier assays. Rescue with wild-type LMX1B and edit candidate ABRA/ARL4C enhancers to distinguish direct effectors from secondary target changes.

Hypothesis: Acute loss of LMX1B in human podocytes directly alters an actin-regulatory transcriptional program before secondary loss of differentiation markers.

Type: acute perturbation, chromatin occupancy, and podocyte functional rescue

Experiment: Introduce endogenous LMX1B interaction-interface mutations and paralog-selective ATG8 knockouts in isogenic human iPSC-derived midbrain dopaminergic neurons; quantify compartment-resolved binding, LMX1B chromatin occupancy, nascent target-gene transcription, autophagic flux, mitochondrial ROS, and rotenone survival, with wild-type and interaction-restoring rescue constructs.

Hypothesis: A defined endogenous ATG8 paralog and compartment-specific interaction with LMX1B are required to couple transcriptional activation to autophagic flux and stress resilience in human midbrain dopaminergic neurons.

Type: endogenous interaction genetics and compartment-resolved functional assay

Experiment: Create isoform-selective splice edits and endogenous epitope tags in matched human podocyte and midbrain dopaminergic-neuron models, then compare localization, DNA occupancy, transcriptomes, LDB1/SSBP3 and ATG8 interactions, and rescue of podocyte or autophagy phenotypes at matched expression levels.

Hypothesis: The three human LMX1B splice isoforms have distinguishable, cell-type-dependent transcriptional and protein-interaction outputs.

Type: isoform-resolved endogenous perturbation and multi-omics comparison

Knowledge Gaps

What is not known β€” curated, literature-grounded statements of the open unknowns (the inverse of core functions).

Gap: The endogenous LMX1B target genes that are necessary and sufficient for human podocyte differentiation and adult maintenance remain unresolved; in particular, COL4A3, COL4A4, NPHS2, and CD2AP should not be treated as a universal direct LMX1B target set.

OPEN BIOLOGY RESIDUAL_SUBGAP

What is known: Mouse null and conditional-knockout studies establish Lmx1b-dependent podocyte differentiation, adult maintenance, and actin-cytoskeleton organization, and human-podocyte assays show LMX1B occupancy at ABRA and ARL4C regulatory regions. However, severely affected human nail-patella syndrome kidneys retained the classic collagen and slit-diaphragm proteins, and adult conditional-knockout work supports an alternative actin-centered mechanism.

Significance: Resolving this target network is necessary to connect LMX1B's established transcription-factor activity to human glomerular disease without overgeneralizing reporter assays or mouse developmental phenotypes.

What would resolve it: Combine acute endogenous LMX1B degradation with CUT&RUN, nascent-transcription profiling, and morphology/filtration-barrier phenotyping in human iPSC-derived podocytes, followed by target-specific rescue and enhancer editing.

Provenance (the field's own admissions):

Gap: It is not known which ATG8-family paralogs are endogenous LMX1B cofactors in human midbrain dopaminergic neurons, how their contributions differ between basal nuclear and starvation-induced cytosolic binding, or what interaction stoichiometry produces the transcriptional effect.

OPEN BIOLOGY RESIDUAL_SUBGAP

What is known: Human HEK293T experiments demonstrate endogenous LMX1B-LC3B co-immunoprecipitation, tagged interaction with all six human ATG8-family proteins, compartment- and nutrient-dependent LC3B binding, and loss of transcriptional and stress-protective output from an LMX1B 308-317 deletion mutant.

Significance: Defining the endogenous cofactor species and compartmental mechanism would explain how ubiquitin-like protein binding modulates LMX1B's core transcriptional activity in the neuronal context most relevant to stress resilience.

What would resolve it: Endogenously tag LMX1B and individual ATG8 paralogs in human iPSC-derived midbrain dopaminergic neurons, then combine paralog-selective perturbation, compartment-resolved interaction assays, LMX1B chromatin occupancy, nascent RNA, autophagic-flux measurements, and rotenone-stress rescue.

Provenance (the field's own admissions):

Gap: Whether the three annotated human LMX1B splice isoforms differ in regulatory-DNA selection, coregulator binding, ATG8 binding, localization, or tissue-specific biological output has not been established.

OPEN BIOLOGY RESIDUAL_SUBGAP

What is known: UniProtKB records three human isoforms, but the current GOA source annotations are not isoform-specific and the direct human autophagy study does not establish an isoform-exclusive function.

Significance: Functional isoform resolution could reveal tissue-selective mechanisms while preventing experimentally tested constructs from being interpreted as proof that a function is unique to one isoform.

What would resolve it: Use isoform-selective splice editing and endogenous tagging in human podocytes and iPSC-derived midbrain dopaminergic neurons, with matched DNA-occupancy, interactome, transcriptome, localization, autophagic-flux, and rescue assays.

Provenance (the field's own admissions):

πŸ“š Additional Documentation

Notes

(LMX1B-notes.md)

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