Total submissions: 3
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Invitae | RCV002042107 | SCV002110659 | uncertain significance | Qualitative or quantitative defects of dysferlin | 2022-02-05 | criteria provided, single submitter | clinical testing | This sequence change falls in intron 25 of the DYSF gene. It does not directly change the encoded amino acid sequence of the DYSF protein. It affects a nucleotide within the consensus splice site. This variant is not present in population databases (gnomAD no frequency). This variant has been observed in individual(s) with limb-girdle muscular dystrophy (PMID: 33610434). Variants that disrupt the consensus splice site are a relatively common cause of aberrant splicing (PMID: 17576681, 9536098). Algorithms developed to predict the effect of sequence changes on RNA splicing suggest that this variant may disrupt the consensus splice site. This variant disrupts the c.2643+5 nucleotide in the DYSF gene. Other variant(s) that disrupt this nucleotide have been determined to be pathogenic (PMID: 27647186; Invitae). This suggests that this nucleotide is clinically significant, and that variants that disrupt this position are likely to be disease-causing. In summary, the available evidence is currently insufficient to determine the role of this variant in disease. Therefore, it has been classified as a Variant of Uncertain Significance. |
Jain Foundation | RCV002042107 | SCV003922049 | likely pathogenic | Qualitative or quantitative defects of dysferlin | 2023-03-13 | criteria provided, single submitter | research | This variant is not present in population databases (gnomAD has no frequency). It has also been identified in one family with dysferlinopathy, segregated with the disease in 2 affected relatives, and was associated with absent dysferlin protein expression (PMID: 36983702). This variant was also found in the heterozygous state in conjunction with another pathogenic DYSF variant (NM_003494.4:c.3113G>A). RNASeq analysis showed that the c.2643+5G>A variant results in the complete skipping of exon 25, which leads to an inframe deletion of 44 amino acids (p.Tyr838_Thr881del; PMID: 36983702). The ACMG classification criteria are: PM2 moderate, PM3 supporting, PM4 moderate, PP1 supporting, PP4 moderate. Based on the above data, this variant has been classified as Likely Pathogenic. |
Neuberg Centre For Genomic Medicine, |
RCV003339763 | SCV004047839 | uncertain significance | Autosomal recessive limb-girdle muscular dystrophy type 2B | criteria provided, single submitter | clinical testing | The splice site variant c.2697+5G>A in DYSF gene has not been reported previously as a pathogenic variant nor as a benign variant, to our knowledge. This variant has been reported to the ClinVar database as a variant of Uncertain Significance with no no functional evidence for this variation. The c.2697+5G>A variant is novel (not in any individuals) in gnomAD Exomes and 1000 Genomes. This variant is close to the canonical splice-site, and the position is strongly conserved. The variant is predicted to affect splicing and loss-offunction is a known mechanism of disease. For these reasons, this variant has been classified as Variant of Uncertain Significance . |