PNPLA3 (Q9NST1) — curation journal

Human PNPLA3 / adiponutrin / iPLA2-epsilon. Patatin-like phospholipase domain-containing
protein 3; closest paralogue of PNPLA2/ATGL. Single-pass type II membrane protein that
partitions between ER/membranes and the lipid droplet (LD) surface. Catalytic dyad
Ser47 (nucleophile) / Asp166 (proton acceptor) in the PNPLA domain (UniProt Q9NST1).

The common missense variant I148M (rs738409) is the single strongest common genetic risk
factor for steatotic liver disease. Three incompatible mechanistic models are currently in
print, and GOA carries annotations that are traceable to two mutually exclusive
biochemical claims. This file records what each primary source actually reports.


1. What has actually been measured with purified protein

TAG lipase / acylglycerol transacylase (Jenkins 2004)

The founding biochemistry. Three iPLA2 family members (adiponutrin/PNPLA3, TTS-2.2, GS2)
expressed in Sf9 cells and affinity purified.
PMID:15364929
PMID:15364929

Note what the abstract does not report: no phospholipase A2 assay result for these three
proteins. The "iPLA2-epsilon"/"phospholipase A2" naming is a family-membership label based on
the dual GXGXXG + GXSXG signature motifs, not a measured PLA2 rate. GOA nevertheless carries
GO:0004623 A2-type glycerophospholipase activity IDA from this paper, and UniProt carries
EC 3.1.1.4 with ECO:0000269|PubMed:15364929. Flagged below as a probable name-driven
over-annotation.

TAG hydrolysis lost in I148M (He 2010)

PMID:20034933
PMID:20034933

Substrate scope, and an explicit negative for acyltransferase (Huang 2011)

PMID:21878620
PMID:21878620
PMID:21878620
PMID:21878620
Full text, results section heading:
PMID:21878620

Curation problem. GOA carries GO:0003841 1-acylglycerol-3-phosphate O-acyltransferase activity with evidence code EXP and PMID:21878620 as the reference, with a plain enables
qualifier (no NOT). That paper reports the opposite: no detectable LPAAT activity, with
CGI-58 as a positive control. The annotation is traceable to UniProt's EC 2.3.1.51 /
RHEA:19709 catalytic-activity block, which tags both PubMed:21878620 and PubMed:22560221 as
ECO:0000269 evidence; only the latter reports a positive result. Either the annotation should
carry a NOT qualifier or the reference is wrong. Recorded in the review and in
suggested_questions; the term itself is kept (see below) because a different paper does
report the activity.

LPAAT (Kumari 2012)

PMID:22560221
PMID:22560221

So two groups, using purified/immunoisolated protein, reach opposite conclusions on the same
reaction, and they also disagree on the direction of the I148M effect (Huang/He: loss of
hydrolase; Kumari: gain of acyltransferase). A protein cannot be the physiologically relevant
catalyst of both the hydrolysis and the synthesis of the same acyl linkage; at most one of
these is the reaction that matters in vivo. GOA currently carries both, which is why the
lipase and acyltransferase branches are adjudicated differently below rather than all
accepted at face value.

Acyl-chain remodelling (Ruhanen 2014)

PMID:24511104
A cell-based readout that is compatible with either a lipase or a transacylase mechanism; it
constrains the net metabolic effect rather than the elementary reaction.

Lipid droplet targeting (Chamoun 2013)

PMID:23398201
PMID:23398201


2. The three current models of I148M

(a) Loss-of-function: PNPLA3 is a PUFA-selective TG lipase feeding VLDL

Johnson et al. 2024, Nat Commun.
PMID:38844467
This paper explicitly frames the open question:
PMID:38844467

(b) Gain-of-function: 148M sequesters ABHD5/CGI-58 away from ATGL

Wang et al. 2025, J Hepatol.
PMID:39550037
PMID:39550037
PMID:39550037
PMID:39550037

Crucially, the same paper is the first to show that PNPLA3's own lipase activity is
ABHD5-stimulated, i.e. it does not deny that PNPLA3 is a lipase:
PMID:39550037
PMID:39550037

The genetic argument against pure LOF, from the same discussion: Pnpla3-/- mice do not
develop steatosis even on high-carbohydrate diets, whereas 148M knock-in mice do, and a
catalytically dead S47A knock-in phenocopies 148M only when the protein is expressed.

(c) Neomorph: 148M blocks both lipolysis and VLDL/ApoB secretion

Sherman et al. 2025, Cell Rep (abstract only in cache).
PMID:41046517
PMID:41046517

Supporting mechanism: LD targeting is required for the ABHD5 interaction

Teskey et al. 2025, J Biol Chem.
PMID:39814233
PMID:39814233

The field acknowledges the conflict

Mikaeeli & Cohen 2025, J Hepatol, is a dedicated editorial titled "Loss or gain of function:
The functional complexity of the PNPLA3 I148M variant" (PMID:39892821). PubMed holds no
abstract for it, so it is cited as evidence that the dispute is recognised rather than quoted.


3. Position taken in this review

Is PNPLA3's physiological molecular function catalytic at all? The honest answer is that
it is both catalytic and non-catalytic, and the non-catalytic arm is the one that carries
the disease signal.

  1. Triacylglycerol lipase activity (GO:0004806) is real and is kept as a core molecular
    function.
    Four independent groups measured TG hydrolysis with purified protein
    (Jenkins 2004, He 2010, Huang 2011, Wang 2025), it is ABHD5-stimulated like ATGL's, and
    Johnson 2024 gives it a substrate preference (polyunsaturated TG) and an in vivo readout.
    What is disputed is not the activity but its quantitative importance: Pnpla3-null mice
    have no hepatic phenotype, so the lipase is dispensable in mouse liver under standard
    conditions.

  2. The ABHD5-competition arm is not represented in GOA at all, and it should be. All
    three 2024-25 models converge on ABHD5/CGI-58 as the node. Wang 2025 shows WT PNPLA3 —
    not only 148M — binds ABHD5 preferentially over ATGL and competes with ATGL for it; 148M
    differs by escaping degradation and accumulating ~40x on LDs, so the same molecular
    activity is simply present in much greater amount. The molecular function is therefore
    sequestration of a lipase co-activator, i.e. inhibition of ATGL. GO:0140311 protein sequestering activity is the closest existing MF term and is used, together with the
    downstream BP GO:0010897 negative regulation of triglyceride catabolic process; both are
    added as action: NEW annotations and as a second core function. Neither GO:0140311 nor
    GO:0055102 lipase inhibitor activity captures "sequesters the co-activator of a lipase",
    so a child term lipase activator sequestering activity is proposed.

  3. The acyltransferase branch is demoted, not deleted. GO:0003841,
    GO:0042171 and GO:0016411 rest on Kumari 2012 (plus Reactome TAS) and are directly
    contradicted by Huang 2011 with an appropriate positive control. Marked
    MARK_AS_OVER_ANNOTATED throughout, with the PMID:21878620 provenance error called out.
    The transacylation terms GO:0051264/GO:0051265 are different and are ACCEPTed: those are
    the CoA-independent mono-olein/diolein transacylase reactions that Jenkins 2004 measured
    directly, and CoA-independent transacylation is chemically compatible with the same Ser47
    nucleophile that performs hydrolysis, so it does not contradict the lipase activity the way
    the acyl-CoA-dependent LPAAT claim does.

  4. A2-type glycerophospholipase (GO:0004623) is marked over-annotated. It is a
    family-name inference; Huang 2011 tested phospholipids and found little or no activity.

  5. Rodent-orthology BP transfers (GO:0009744 response to sucrose, GO:0032869 cellular
    response to insulin stimulus, GO:0050872 white fat cell differentiation, GO:1905243
    response to T3) describe transcriptional regulation of PNPLA3 by nutritional state, not
    processes PNPLA3 carries out; they are kept as non-core, except GO:0050872 white fat cell differentiation, which is marked over-annotated (PNPLA3 is induced during adipogenesis, but
    there is no evidence it drives it). UniProt records the same:
    "By changes in energy balance: down-regulated following very low-calorie diet, whereas
    refeeding elevates the mRNA level" (Q9NST1 INDUCTION). Demoted to non-core.

4. Open questions carried into the review