PPP1R21

Chr 2AR

protein phosphatase 1 regulatory subunit 21

Also known as: CCDC128, FERRY2, Fy-2, KLRAQ1, NEDHFBA

PPP1R21 encodes a component of the FERRY complex that binds mRNA and mediates endosomal transport of specific mRNAs and ribosomes to early endosomes. Mutations cause autosomal recessive neurodevelopmental disorder with hypotonia, facial dysmorphism, and brain abnormalities. The gene shows low constraint to loss-of-function variants (pLI nearly zero), consistent with its recessive inheritance pattern.

Summary from RefSeq, OMIM, UniProt
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Primary Disease Associations & Inheritance

Neurodevelopmental disorder with hypotonia, facial dysmorphism, and brain abnormalitiesMIM #619383
AR
0
Active trials
9
Pubs (1 yr)
37
P/LP submissions
6%
P/LP missense
0.69
LOEUF
LOF
Mechanism· G2P
Clinical SummaryPPP1R21
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Gene-Disease Validity (ClinGen)
neurodevelopmental disorder with hypotonia, facial dysmorphism, and brain abnormalities · ARStrong

Strong evidence — appropriate for clinical testing

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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ClinVar Variants
34 unique Pathogenic / Likely Pathogenic· 159 VUS of 261 total submissions
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GeneReview available — PPP1R21
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.69LOEUF
pLI 0.000
Z-score 3.37
OE 0.49 (0.360.69)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
-1.92Z-score
OE missense 1.27 (1.181.37)
506 obs / 398.3 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.49 (0.360.69)
00.351.4
Missense OE1.27 (1.181.37)
00.61.4
Synonymous OE1.10
01.21.6
LoF obs/exp: 25 / 50.9Missense obs/exp: 506 / 398.3Syn Z: -0.99
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitivePPP1R21-related neurodevelopmental disorderLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.7033th %ile
GOF
0.5268th %ile
LOF
0.4136th %ile

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

261 submitted variants in ClinVar

Classification Summary

Pathogenic25
Likely Pathogenic9
VUS159
Likely Benign32
Benign5
Conflicting2
25
Pathogenic
9
Likely Pathogenic
159
VUS
32
Likely Benign
5
Benign
2
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
6
2
17
0
25
Likely Pathogenic
3
0
6
0
9
VUS
0
149
10
0
159
Likely Benign
0
8
4
20
32
Benign
0
0
2
3
5
Conflicting
2
Total91593923232

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

PPP1R21 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →
Clinical Literature
Open Research Assistant →
Recent Gene-Specific Literature
Gene in title · MEDLINE · newest first
Europe PMC