Total submissions: 2
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
Revvity Omics, |
RCV000770936 | SCV004238087 | uncertain significance | Triosephosphate isomerase deficiency | 2023-05-10 | criteria provided, single submitter | clinical testing | |
Palladino Lab, |
RCV000770936 | SCV000882820 | pathogenic | Triosephosphate isomerase deficiency | no assertion criteria provided | research | The R190Q variant in TPI1 had not been previously reported, yet we find this in a compound heterozygous patient (TPI1[E104D]/[R190Q]) suffering from severe hemolytic anemia, cerebral atrophy and periventricular leukomalacia, neuromuscular impairment with respiratory deficiency, bilateral diaframatic paralysis. TPI[E104D] is known to cause TPI deficiency in homozygous or compound heterozygous patients (PMID: 9338582), and here we have shown that R189 mutations alters the structure of the enzyme's catalytic site, and reduces protein levels in animal models and patient fibroblasts. |