Search GLEAM-DB / CoGenEx-PM3

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Input query
Recognized gene
GUSB
Normalized c.HGVS
c.-10A>T, c.1091C>T, c.1138G>A, c.1222C>T, c.1244C>T and 19 more
Normalized p.HGVS
p.(=), p.(Ala380Thr), p.(Ala442Thr), p.(Ala442Val), p.(Arg116His) and 18 more
Matching records
57
PM3-positive records
14

Results are ordered by PM3-relevant evidence first. Reviewed source only indicates whether main text, supplementary material, or both were reviewed; it is not the evidence conclusion.

Download current query results as TSV
Gene Variant Evidence PM3 context Publication Reviewed source Actions
GUSB NM_000181.4:c.1091C>T Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.104C>A; Ser35Ter
context: Compound heterozygous candidate
40640912
Lysosomal storage disorders in nonimmune hydrops fetalis diagnosed by exome sequencing.
Orphanet journal of rare diseases, 2025
Main article
Open
GUSB NM_000181.4:c.1324G>A Phase-unconfirmed biallelic evidence
High confidence
Homozygous for query variant
context: Homozygous evidence
40640912
Lysosomal storage disorders in nonimmune hydrops fetalis diagnosed by exome sequencing.
Orphanet journal of rare diseases, 2025
Main article
Open
GUSB NM_000181.4:c.1729C>T Phase-unconfirmed biallelic evidence
High confidence
Homozygous for query variant
context: Homozygous evidence
40640912
Lysosomal storage disorders in nonimmune hydrops fetalis diagnosed by exome sequencing.
Orphanet journal of rare diseases, 2025
Main article
Open
GUSB NM_000181.4:c.380A>G Phase-unconfirmed biallelic evidence
Needs review
Possible compound heterozygous with Leu322Phe
context: Compound heterozygous candidate
38715031
Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.
Orphanet journal of rare diseases, 2024
Main article
Open
GUSB NM_000181.4:c.1324G>A Phase-unconfirmed biallelic evidence
High confidence
Homozygous for query variant
context: Homozygous evidence
38149215
A de novo homozygous missense mutation of the GUSB gene leads to mucopolysaccharidosis type VII identification in a family with twice adverse pregnancy outcomes due to non-immune hydrops fetalis.
Molecular genetics and metabolism reports, 2023
Main article
Open
GUSB NM_000181.4:c.1222C>T Phase-unconfirmed biallelic evidence
High confidence
Potential PM3 evidence identified; genotype context not structured
context: PM3 evidence context not structured
36578769
Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome).
Therapeutics and clinical risk management, 2022
Main article
Open
GUSB NM_000181.4:c.1325C>T Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.50G > C; p.W17S
context: Compound heterozygous candidate
34686181
Description of the molecular and clinical characteristics of the mucopolysaccharidosis type VII Iberian cohort.
Orphanet journal of rare diseases, 2021
Main article
Open
GUSB NM_000181.4:c.1091C>T Phase-unconfirmed biallelic evidence
High confidence
Confirmed in trans with c.104C > A; p.Ser35*
context: Confirmed in trans
33897756
Value of Exome Sequencing in Diagnosis and Management of Recurrent Non-immune Hydrops Fetalis: A Retrospective Analysis.
Frontiers in genetics, 2021
Main article
Open
GUSB NM_000181.4:c.1325C>T Phase-unconfirmed biallelic evidence
Needs review
Possible compound heterozygous with p.P67L
context: Compound heterozygous candidate
31732130
Newborn Screening for Mucopolysaccharidoses: Results of a Pilot Study with 100 000 Dried Blood Spots.
The Journal of pediatrics, 2020
Main article
Open
GUSB NM_000181.4:c.1222C>T Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with P415L
context: Compound heterozygous candidate
26908836
Clinical course of sly syndrome (mucopolysaccharidosis type VII).
Journal of medical genetics, 2016
Main article
Open
GUSB NM_000181.4:c.1244C>T Phase-unconfirmed biallelic evidence
Needs review
Possible compound heterozygous with p.P408S
context: Compound heterozygous candidate
26908836
Clinical course of sly syndrome (mucopolysaccharidosis type VII).
Journal of medical genetics, 2016
Main article
Open
GUSB NM_000181.4:c.266A>G Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with p.D362N
context: Compound heterozygous candidate
26908836
Clinical course of sly syndrome (mucopolysaccharidosis type VII).
Journal of medical genetics, 2016
Main article
Open
GUSB NM_000181.4:c.1222C>T Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.1244C>T; p.P415L
context: Compound heterozygous candidate
19224584
Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome).
Human mutation, 2009
Main article
Open
GUSB NM_000181.4:c.1244C>T Phase-unconfirmed biallelic evidence
High confidence
Possible compound heterozygous with c.1222C>T; p.P408S; p.P415L
context: Compound heterozygous candidate
19224584
Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome).
Human mutation, 2009
Main article
Open
GUSB NM_000181.4:c.1091C>T No PM3 Evidence Identified
Not assessed
No PM3 candidate genotype identified 41149794
Autism Spectrum Disorder: The Cerebellum, Genes, and Pathways.
Neurology international, 2025
Main article
Open
GUSB NM_000181.4:c.1222C>T No PM3 Evidence Identified
Not assessed
No PM3 candidate genotype identified 40275366
Analysis of genomic ancestry and characterization of a new variant in MPS type VII.
Orphanet journal of rare diseases, 2025
Main article
Open
GUSB NM_000181.4:c.1244C>T No PM3 Evidence Identified
Not assessed
No PM3 candidate genotype identified 36578769
Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome).
Therapeutics and clinical risk management, 2022
Main article
Open
GUSB NM_000181.4:c.-10A>T No PM3 Evidence Identified
Not assessed
No PM3 candidate genotype identified 35419288
Case Report: A Novel Pathomechanism in PEComa by the Loss of Heterozygosity of TP53.
Frontiers in oncology, 2022
Main article and supplement
Open
GUSB NM_000181.4:c.493C>T No PM3 Evidence Identified
Not assessed
No PM3 candidate genotype identified 35229492
Systematic review and meta-analysis of genomic alterations in acral melanoma.
Pigment cell & melanoma research, 2022
Main article
Open
GUSB NM_000181.4:c.695C>G No PM3 Evidence Identified
Not assessed
No PM3 candidate genotype identified 33628140
Technological Advancements in Monoclonal Antibodies.
TheScientificWorldJournal, 2021
Main article
Open