A cis-regulatory site downregulates PTHLH in translocation t(8;12)(q13;p11.2) and leads to Brachydactyly Type E

PG Maass, J Wirth, A Aydin, A Rump… - Human molecular …, 2010 - academic.oup.com
PG Maass, J Wirth, A Aydin, A Rump, S Stricker, S Tinschert, M Otero, K Tsuchimochi…
Human molecular genetics, 2010academic.oup.com
Parathyroid hormone-like hormone (PTHLH) is an important chondrogenic regulator;
however, the gene has not been directly linked to human disease. We studied a family with
autosomal-dominant Brachydactyly Type E (BDE) and identified at (8; 12)(q13; p11. 2)
translocation with breakpoints (BPs) upstream of PTHLH on chromosome 12p11. 2 and a
disrupted KCNB2 on 8q13. We sequenced the BPs and identified a highly conserved
Activator protein 1 (AP-1) motif on 12p11. 2, together with a C-ets-1 motif translocated from …
Abstract
Parathyroid hormone-like hormone (PTHLH) is an important chondrogenic regulator; however, the gene has not been directly linked to human disease. We studied a family with autosomal-dominant Brachydactyly Type E (BDE) and identified a t(8;12)(q13;p11.2) translocation with breakpoints (BPs) upstream of PTHLH on chromosome 12p11.2 and a disrupted KCNB2 on 8q13. We sequenced the BPs and identified a highly conserved Activator protein 1 (AP-1) motif on 12p11.2, together with a C-ets-1 motif translocated from 8q13. AP-1 and C-ets-1 bound in vitro and in vivo at the derivative chromosome 8 breakpoint [der(8) BP], but were differently enriched between the wild-type and BP allele. We differentiated fibroblasts from BDE patients into chondrogenic cells and found that PTHLH and its targets, ADAMTS-7 and ADAMTS-12 were downregulated along with impaired chondrogenic differentiation. We next used human and murine chondrocytes and observed that the AP-1 motif stimulated, whereas der(8) BP or C-ets-1 decreased, PTHLH promoter activity. These results are the first to identify a cis-directed PTHLH downregulation as primary cause of human chondrodysplasia.
Oxford University Press