Wiskott–Aldrich syndrome: Oral findings and microbiota in children and review of the literature
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DOI: 10.1002/cre2.503
Abstract: Wiskott–Aldrich syndrome (WAS) is a rare X‐linked primary immunodeficiency, characterized by micro‐thrombocytopenia, recurrent infections, and eczema. This study aims to describe common oral manifestations and evaluate oral microbioma of WAS patients. read more here.
Keywords: aldrich syndrome; oral findings; findings microbiota; wiskott aldrich ... See more keywords
IL‐18: A potential inflammation biomarker in Wiskott–Aldrich syndrome
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DOI: 10.1002/eji.202049024
Abstract: Analysis of serum cytokine levels in Wiskott–Aldrich syndrome patients pre‐ and post‐ treatment reveals IL‐18 as a stable and reliable marker of inflammation. Definitive stem cell treatment with good myeloid correction correlates with resolution of… read more here.
Keywords: potential inflammation; inflammation biomarker; biomarker wiskott; inflammation ... See more keywords
Phase I trial of low-dose interleukin 2 therapy in patients with Wiskott-Aldrich syndrome.
Sign Up to like & getrecommendations! Published in 2017 at "Clinical immunology"
DOI: 10.1016/j.clim.2017.02.001
Abstract: BACKGROUND Low dose IL-2 can restore the function of T and NK cells from Wiskott-Aldrich (WAS) patients. However, the safety of in vivo IL-2 in WAS is unknown. OBJECTIVES A phase-I study to assess safety… read more here.
Keywords: therapy; low dose; day dose; dose ... See more keywords
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome
Sign Up to like & getrecommendations! Published in 2022 at "Nature Medicine"
DOI: 10.1038/s41591-021-01641-x
Abstract: Patients with Wiskott–Aldrich syndrome (WAS) lacking a human leukocyte antigen-matched donor may benefit from gene therapy through the provision of gene-corrected, autologous hematopoietic stem/progenitor cells. Here, we present comprehensive, long-term follow-up results (median follow-up, 7.6 years)… read more here.
Keywords: long term; gene therapy; gene; wiskott aldrich ... See more keywords
Wiskott–Aldrich syndrome: Two case reports with a novel mutation
Sign Up to like & getrecommendations! Published in 2017 at "Pediatric Hematology and Oncology"
DOI: 10.1080/08880018.2017.1397072
Abstract: ABSTRACT Background: The Wiskott–Aldrich syndrome (WAS) is X-linked recessive disorder associated with microplatelet thrombocytopenia, eczema, infections, and an increased risk of autoimmunity and lymphoid neoplasia. The originally described features of WAS include susceptibility to infections,… read more here.
Keywords: wiskott aldrich; aldrich syndrome; mutation; case ... See more keywords
Wiskott–Aldrich syndrome in a child presenting with macrothrombocytopenia
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DOI: 10.1080/09537104.2016.1246715
Abstract: Abstract Wiskott–Aldrich syndrome (WAS) is a rare X-linked recessive disease resulting from variants in the WAS gene, characterized by a triad of immunodeficiency, eczema, and thrombocytopenia. Despite the fact that WAS is traditionally differentiated from… read more here.
Keywords: presenting macrothrombocytopenia; child presenting; aldrich syndrome; wiskott aldrich ... See more keywords
Report of clinical presentations and two novel mutations in patients with Wiskott-Aldrich syndrome/X-linked Thrombocytopenia.
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DOI: 10.1080/09537104.2021.1988549
Abstract: Wiskott-Aldrich syndrome (WAS)/X-linked thrombocytopenia (XLT) is a rare X-linked disease characterized by thrombocytopenia, eczema, and recurrent infection. In addition, WAS/XLT increases incidence of autoimmune diseases and malignancies. We reported 7 male patients, 2 with WAS… read more here.
Keywords: novel mutations; syndrome linked; aldrich syndrome; two novel ... See more keywords
Comparison of Zinc Finger Nucleases Versus CRISPR-Specific Nucleases for Genome Editing of the Wiskott-Aldrich Syndrome Locus.
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DOI: 10.1089/hum.2017.047
Abstract: Primary immunodeficiencies, including Wiskott-Aldrich syndrome (WAS), are a main target for genome-editing strategies using specific nucleases (SNs) because a small number of corrected hematopoietic stem cells could cure patients. In this work, we have designed… read more here.
Keywords: crispr; crispr cas9; delivery; zfns ... See more keywords
Wiskott-Aldrich Syndrome Misdiagnosed as Immune Thrombocytopenic Purpura: A Case Report.
Sign Up to like & getrecommendations! Published in 2018 at "Journal of Pediatric Hematology/Oncology"
DOI: 10.1097/mph.0000000000000949
Abstract: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency characterized by various clinical phenotypes. We report the case of a 3-year-old immigrant boy presenting with persistent infant-onset thrombocytopenia treated for refractory immune thrombocytopenic purpura. Sequence analysis… read more here.
Keywords: wiskott aldrich; thrombocytopenic purpura; aldrich syndrome; immune thrombocytopenic ... See more keywords
Successful Haploidentical Stem Cell Transplant With Posttransplant Cyclophosphamide in Wiskott-Aldrich Syndrome With Myeloablative Conditioning.
Sign Up to like & getrecommendations! Published in 2020 at "Journal of Pediatric Hematology/Oncology"
DOI: 10.1097/mph.0000000000001841
Abstract: Hematopoietic stem cell transplant (HSCT) is the only curative treatment modality for Wiskott-Aldrich syndrome. Haploidentical HSCT with posttransplant cyclophosphamide (PTCy) is an upcoming option in children with nonmalignant conditions. However, only few cases have been… read more here.
Keywords: aldrich syndrome; myeloablative conditioning; hsct; wiskott aldrich ... See more keywords
Acquired Thrombotic Thrombocytopenic Purpura in a 5-Year-old Child With Wiskott-Aldrich Syndrome
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DOI: 10.1097/mph.0000000000002150
Abstract: Thrombocytopenia is often seen as a laboratory finding during childhood. A supposed idiopathic thrombocytopenic purpura patient who was later diagnosed as Wiskott-Aldrich syndrome (WAS) and developed acquired thrombotic thrombocytopenic purpura (aTTP). Although autoimmune manifestations in… read more here.
Keywords: acquired thrombotic; thrombocytopenic purpura; aldrich syndrome; year ... See more keywords