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Repeat Disorders v1.11 TBC1D7_OPDM_CGG Bryony Thompson Marked STR: TBC1D7_OPDM_CGG as ready
Repeat Disorders v1.11 TBC1D7_OPDM_CGG Bryony Thompson Str: tbc1d7_opdm_cgg has been classified as Amber List (Moderate Evidence).
Repeat Disorders v1.11 TBC1D7_OPDM_CGG Bryony Thompson Classified STR: TBC1D7_OPDM_CGG as Amber List (moderate evidence)
Repeat Disorders v1.11 TBC1D7_OPDM_CGG Bryony Thompson Str: tbc1d7_opdm_cgg has been classified as Amber List (Moderate Evidence).
Repeat Disorders v1.10 TBC1D7_OPDM_CGG Bryony Thompson edited their review of STR: TBC1D7_OPDM_CGG: Changed rating: AMBER
Repeat Disorders v1.10 TBC1D7_OPDM_CGG Bryony Thompson Classified STR: TBC1D7_OPDM_CGG as Green List (high evidence)
Repeat Disorders v1.10 TBC1D7_OPDM_CGG Bryony Thompson Str: tbc1d7_opdm_cgg has been classified as Green List (High Evidence).
Repeat Disorders v1.9 TBC1D7_OPDM_CGG Bryony Thompson STR: TBC1D7_OPDM_CGG was added
STR: TBC1D7_OPDM_CGG was added to Repeat Disorders. Sources: Literature
Mode of inheritance for STR: TBC1D7_OPDM_CGG was set to MONOALLELIC, autosomal or pseudoautosomal, NOT imprinted
Publications for STR: TBC1D7_OPDM_CGG were set to 41959811
Phenotypes for STR: TBC1D7_OPDM_CGG were set to Oculopharyngodistal myopathy, TBC1D7-related MONDO:0025193
Review for STR: TBC1D7_OPDM_CGG was set to GREEN
Added comment: Preprint PMID 41959811 reports 3 families with a heterozygous 5'UTR CCG expansion in TBC1D7 and oculopharyngodistal myopathy. Affected individuals had 83, 87, 113, 137 and 148 repeats (n=5). All 3 families share a core 16 kb haplotype, consistent with a common ancestral origin. An unaffected transmitting father carries the largest allele, 184 repeats, hypermethylated. Gain of function is the proposed mechanism of disease. Patient-derived fibroblasts show increased TBC1D7 expression, and muscle biopsy shows p62-positive intranuclear inclusions, supporting a dominant toxic gain-of-function mechanism analogous to other CCG-expansion disorders. No normal range is defined. 79/70,752 GE control alleles had >50 repeats.
The reference and 94.3% of 1,718 control alleles carry an interrupted CCGCTG structure, while patient alleles are long pure CCG.
Sources: Literature