Germline NLRP1 Mutations Cause Skin Inflammatory and Cancer Susceptibility Syndromes via Inflammasome Activation

نویسندگان

  • Franklin L. Zhong
  • Ons Mamaï
  • Lorenzo Sborgi
  • Lobna Boussofara
  • Richard Hopkins
  • Kim Robinson
  • Ildikó Szeverényi
  • Takuya Takeichi
  • Reshmaa Balaji
  • Aristotle Lau
  • Hazel Tye
  • Keya Roy
  • Carine Bonnard
  • Patricia J. Ahl
  • Leigh Ann Jones
  • Paul J. Baker
  • Lukas Lacina
  • Atsushi Otsuka
  • Pierre R. Fournie
  • François Malecaze
  • E. Birgitte Lane
  • Masashi Akiyama
  • Kenji Kabashima
  • John E. Connolly
  • Seth L. Masters
  • Vincent J. Soler
  • Salma Samir Omar
  • John A. McGrath
  • Roxana Nedelcu
  • Moez Gribaa
  • Mohamed Denguezli
  • Ali Saad
  • Sebastian Hiller
  • Bruno Reversade
چکیده

Inflammasome complexes function as key innate immune effectors that trigger inflammation in response to pathogen- and danger-associated signals. Here, we report that germline mutations in the inflammasome sensor NLRP1 cause two overlapping skin disorders: multiple self-healing palmoplantar carcinoma (MSPC) and familial keratosis lichenoides chronica (FKLC). We find that NLRP1 is the most prominent inflammasome sensor in human skin, and all pathogenic NLRP1 mutations are gain-of-function alleles that predispose to inflammasome activation. Mechanistically, NLRP1 mutations lead to increased self-oligomerization by disrupting the PYD and LRR domains, which are essential in maintaining NLRP1 as an inactive monomer. Primary keratinocytes from patients experience spontaneous inflammasome activation and paracrine IL-1 signaling, which is sufficient to cause skin inflammation and epidermal hyperplasia. Our findings establish a group of non-fever inflammasome disorders, uncover an unexpected auto-inhibitory function for the pyrin domain, and provide the first genetic evidence linking NLRP1 to skin inflammatory syndromes and skin cancer predisposition.

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عنوان ژورنال:
  • Cell

دوره 167  شماره 

صفحات  -

تاریخ انتشار 2016