Total submissions: 1
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Kids Neuroscience Centre, |
RCV001726521 | SCV001571534 | likely pathogenic | Hereditary spastic paraplegia 4 | no assertion criteria provided | clinical testing | Detected one abnormal splicing event, out-of-frame exon 16 duplication (r.1688_1728dup). This event causes a frameshift encoding 1 missense amino acid and a premature termination codon (p.(Met577Asnfs*2)). These transcripts are not predicted to be targeted by NMD as the premature termination codon is encoded 35 bp upstream from the last exon-exon junction. SPAST transcripts with exon 16 duplication encode spastin protein lacking 40 evolutionarily conserved amino acids from the spastin domain. Missense substitutions p.(Met577Arg), p.(Ser588Pro) and p.(Ile592Lys) within thi s region of the spastin domain are described as likely pathogenic variants in ClinVar. Therefore, it is the opinion of this laboratory that loss of 40 amino acids p.(Met577_Val616del) from the conserved spastin domain is consistent with likely non/dysfunc tion of the encoded spastin protein. |