Treatment of severe forms of LPS-responsive beige-like anchor protein deficiency with allogeneic hematopoietic stem cell transplantation.
Seidel, Markus G; Böhm, Katrin; Dogu, Figen; Worth, Austen; Thrasher, Adrian; Florkin, Benoit; Ikinciogullari, Aydan; Peters, Anke; Bakhtiar, Shahrzad; Meeths, Marie; Stepensky, Polina; Meyts, Isabelle; Sharapova, Svetlana O; Gámez-Díaz, Laura; Hammarström, Lennart; Ehl, Stephan; Grimbacher, Bodo; Gennery, Andrew R.
J Allergy Clin Immunol
; 141(2): 770-775.e1, 2018 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-28502825
New therapeutic approach by sirolimus for enteropathy treatment in patients with LRBA deficiency.
Successful Hematopoietic Stem Cell Transplantation in a Patient with LPS-Responsive Beige-Like Anchor (LRBA) Gene Mutation.
The extended phenotype of LPS-responsive beige-like anchor protein (LRBA) deficiency.
Multiple Presentations of LRBA Deficiency: a Single-Center Experience.
Polyautoimmunity in Patients with LPS-Responsive Beige-Like Anchor (LRBA) Deficiency.
Clinical, immunologic, molecular analyses and outcomes of iranian patients with LRBA deficiency: A longitudinal study.
Respiratory manifestations in LPS-responsive beige-like anchor (LRBA) protein-deficient patients.
Enhanced susceptibility to chemically induced colitis caused by excessive endosomal TLR signaling in LRBA-deficient mice.
Two male siblings with a novel mutation presenting with different findings of IPEX syndrome.
Tregopathies: Monogenic diseases resulting in regulatory T-cell deficiency.