Total submissions: 7
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Laboratory for Molecular Medicine, |
RCV000825543 | SCV000966860 | likely pathogenic | Primary ciliary dyskinesia | 2018-08-23 | criteria provided, single submitter | clinical testing | The p.Ser39X variant in RSPH4A has been reported in one compound heterozygous in dividual and one heterozygous individual without a second variant identified, bo th with primary ciliary dyskinesia (Daniels et al, 2013). This variant has been identified in 5/126582 European chromosomes by the Genome Aggregation Database ( gnomAD, http://gnomad.broadinstitute.org). This nonsense variant leads to a prem ature termination codon at position 39 which is predicted to lead to a truncated or absent protein. Bialleic loss of function of the RSPH4A gene has been associ ated with primary ciliary dyskinesia. Although this variant has been seen in the general population, its frequency is low enough to be consistent with a recessi ve carrier frequency. In summary, although additional studies are required to fu lly establish its clinical significance, the p.Ser39X variant in RSPH4A is likel y pathogenic for primary ciliary dyskinesia. ACMG/AMP Criteria applied: PVS1_Str ong, PM2, PM#_Supporting. |
UNC Molecular Genetics Laboratory, |
RCV000825543 | SCV001431717 | pathogenic | Primary ciliary dyskinesia | 2018-04-26 | criteria provided, single submitter | clinical testing | |
Johns Hopkins Genomics, |
RCV002282387 | SCV002570368 | pathogenic | Primary ciliary dyskinesia 11 | 2022-08-03 | criteria provided, single submitter | clinical testing | This RSPH4A nonsense variant has been observed in the compound heterozygous state in one individual with primary ciliary dyskinesia, and as a single heterozygous variant without a second variant identified in another unrelated individual with primary ciliary dyskinesia. This variant (rs368110732) is rare (<0.1%) in a large population dataset (gnomAD v2.1.1: 5/282514 total alleles; 0.0018%; no homozygotes), and has been reported in ClinVar (Variation ID 666983). This nonsense variant results in a premature stop codon in exon 1, likely leading to nonsense-mediated decay and lack of protein production. We consider c.116C>A;p.Ser39Ter in RSPH4A to be pathogenic. |
Ambry Genetics | RCV000825543 | SCV002627023 | pathogenic | Primary ciliary dyskinesia | 2017-01-13 | criteria provided, single submitter | clinical testing | The p.S39* pathogenic mutation (also known as c.116C>A), located in coding exon 1 of the RSPH4A gene, results from a C to A substitution at nucleotide position 116. This changes the amino acid from a serine to a stop codon within coding exon 1. This mutation was described in individuals with primary ciliary dyskinesia, including one individual with a second alteration confirmed in trans (Daniels ML et al. Hum. Mutat., 2013 Oct;34:1352-6). In addition to the clinical data presented in the literature, this alteration is expected to result in loss of function by premature protein truncation or nonsense-mediated mRNA decay. As such, this alteration is interpreted as a disease-causing mutation. |
Fulgent Genetics, |
RCV002282387 | SCV002809948 | pathogenic | Primary ciliary dyskinesia 11 | 2022-04-06 | criteria provided, single submitter | clinical testing | |
Labcorp Genetics |
RCV000825543 | SCV003279219 | pathogenic | Primary ciliary dyskinesia | 2025-01-28 | criteria provided, single submitter | clinical testing | This sequence change creates a premature translational stop signal (p.Ser39*) in the RSPH4A gene. It is expected to result in an absent or disrupted protein product. Loss-of-function variants in RSPH4A are known to be pathogenic (PMID: 19200523). This variant is present in population databases (rs368110732, gnomAD 0.004%). This premature translational stop signal has been observed in individual(s) with primary ciliary dyskinesia (PMID: 23798057). ClinVar contains an entry for this variant (Variation ID: 666983). For these reasons, this variant has been classified as Pathogenic. |
Mayo Clinic Laboratories, |
RCV004792546 | SCV005413748 | pathogenic | not provided | 2024-01-03 | criteria provided, single submitter | clinical testing | PP4, PM3_strong, PVS1 |