Identical ift140 variants cause variable skeletal ciliopathy phenotypes challenges for the accurate diagnosis

HIGHLIGHTS

SUMMARY

    Variants in IFT140 are implicated in the pathogenesis of CED and MZSDS, but also in JATD, Bardet-Biedl syndrome (BBS), Optiz trigonocephaly C syndrome (OTCS), and isolated retinitis pigmentosa (RP) displaying the complexity of ciliopathies (Cole and Snell, 2009; Schmidts et_al, 2013; Bifari et_al, 2016; Schaefer et_al, 2016; Pena-Padilla et_al, 2017). Dysfunctional IFT140 results in an accumulation of IFT proteins at the ciliary tip and in cilium shortening (Piperno et_al, 1998; Miller et_al, 2013). The cilium length was measured using the ALPACA tool, described in detail by Doornbos et_al, based on the length . . .

     

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