| Literature DB >> 31195081 |
Maria E Maccari1, Carsten Speckmann1, Maximilian Heeg1, Antonia Reimer2, Federica Casetti2, Cristina Has2, Stephan Ehl1, Carla N Castro3.
Abstract
This study reports a patient with severe skin disease in the context of profound immunodeficiency explained by two concomitant genetic diseases caused by two novel homozygous loss-of-function mutations in PLEC1 and CARMIL2. The work provides additional information on the clinical and immunological manifestations of CARMIL2 deficiency and highlights the particular diagnostic and therapeutic challenge represented by the concomitant presence of two rare monogenic disorders.Entities:
Keywords: CARMIL2; Epidermolysis bullosa; Immunodeficiency; Plectin; RLTPR; Whole exome sequencing
Mesh:
Substances:
Year: 2019 PMID: 31195081 DOI: 10.1016/j.clim.2019.06.004
Source DB: PubMed Journal: Clin Immunol ISSN: 1521-6616 Impact factor: 3.969