MBTPS1

Chr 16

membrane bound transcription factor peptidase, site 1

Also known as: CAOP, PCSK8, S1P, SEDKF, SKI-1

This gene encodes a member of the subtilisin-like proprotein convertase family, which includes proteases that process protein and peptide precursors trafficking through regulated or constitutive branches of the secretory pathway. The encoded protein undergoes an initial autocatalytic processing event in the ER to generate a heterodimer which exits the ER and sorts to the cis/medial-Golgi where a second autocatalytic event takes place and the catalytic activity is acquired. It encodes a type 1 membrane bound protease which is ubiquitously expressed and regulates cholesterol or lipid homeostasis via cleavage of substrates at non-basic residues. Mutations in this gene may be associated with lysosomal dysfunction. [provided by RefSeq, Feb 2014]

GeneReviewsResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

UniProtSpondyloepiphyseal dysplasia, Kondo-Fu type
UniProtCataract, alopecia, oral mucosal disorder, and psoriasis-like syndrome

Clinical highlights

Gene-disease validity (ClinGen)
spondyloepiphyseal dysplasia, kondo-fu type · ARDefinitivesufficient evidence for diagnostic panels
0
Active trials
12
Pubs (1 yr)
P/LP submissions
P/LP missense
0.59
LOEUF
DN
Mechanism· predicted
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GeneReview available — MBTPS1
Authoritative clinical overview · Recommended first read
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Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint?
0.59LOEUF
pLI 0.000
Z-score 4.08
OE 0.42 (0.300.59)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint?
-1.45Z-score
OE missense 1.16 (1.091.24)
715 obs / 613.8 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios?
LoF OE?0.42 (0.300.59)
00.351.4
Missense OE?1.16 (1.091.24)
00.61.4
Synonymous OE?1.20
01.21.6
LoF obs/exp: 24 / 57.4Missense obs/exp: 715 / 613.8Syn Z: -2.42

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

MBTPS1 · 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 →