{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T18:45:19Z","timestamp":1779129919125,"version":"3.51.4"},"reference-count":44,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2019,11,5]],"date-time":"2019-11-05T00:00:00Z","timestamp":1572912000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Pediatr."],"DOI":"10.3389\/fped.2019.00451","type":"journal-article","created":{"date-parts":[[2019,11,5]],"date-time":"2019-11-05T00:50:44Z","timestamp":1572915044000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":22,"title":["Wiskott-Aldrich Syndrome (WAS) and Dedicator of Cytokinesis 8- (DOCK8) Deficiency"],"prefix":"10.3389","volume":"7","author":[{"given":"Michael H.","family":"Albert","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alexandra F.","family":"Freeman","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1965","published-online":{"date-parts":[[2019,11,5]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1007\/s10875-017-0453-z","article-title":"Clinical manifestations and pathophysiological mechanisms of the wiskott-aldrich syndrome","volume":"38","author":"Candotti","year":"2018","journal-title":"J Clin Immunol."},{"key":"B2","doi-asserted-by":"publisher","first-page":"1015","DOI":"10.1586\/1744666X.2015.1062366","article-title":"Current and emerging treatment options for Wiskott-Aldrich syndrome","volume":"11","author":"Worth","year":"2015","journal-title":"Expert Rev Clin Immunol."},{"key":"B3","doi-asserted-by":"publisher","first-page":"1857","DOI":"10.1002\/eji.201646715","article-title":"Wiskott-Aldrich syndrome protein: emerging mechanisms in immunity","volume":"47","author":"Rivers","year":"2017","journal-title":"Eur J Immunol"},{"key":"B4","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1097\/MOH.0b013e32834114bc","article-title":"Clinical spectrum, pathophysiology and treatment of the Wiskott-Aldrich syndrome","volume":"18","author":"Albert","year":"2011","journal-title":"Curr Opin Hematol."},{"key":"B5","doi-asserted-by":"publisher","first-page":"3231","DOI":"10.1182\/blood-2009-09-239087","article-title":"X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options","volume":"115","author":"Albert","year":"2010","journal-title":"Blood"},{"key":"B6","doi-asserted-by":"publisher","first-page":"1510","DOI":"10.1182\/blood-2012-08-448118","article-title":"Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome","volume":"121","author":"Mahlaoui","year":"2013","journal-title":"Blood"},{"key":"B7","doi-asserted-by":"publisher","first-page":"1364","DOI":"10.1016\/S0140-6736(68)92672-X","article-title":"Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome","volume":"2","author":"Bach","year":"1968","journal-title":"Lancet"},{"key":"B8","doi-asserted-by":"publisher","first-page":"1675","DOI":"10.1182\/blood-2010-11-319376","article-title":"Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980\u20132009: an international collaborative study","volume":"118","author":"Moratto","year":"2011","journal-title":"Blood"},{"key":"B9","doi-asserted-by":"publisher","first-page":"439","DOI":"10.1182\/blood-2007-03-076679","article-title":"Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation","volume":"111","author":"Ozsahin","year":"2008","journal-title":"Blood"},{"key":"B10","doi-asserted-by":"publisher","first-page":"1598","DOI":"10.1182\/blood.V97.6.1598","article-title":"Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program","volume":"97","author":"Filipovich","year":"2001","journal-title":"Blood"},{"key":"B11","doi-asserted-by":"publisher","first-page":"671","DOI":"10.1038\/sj.bmt.1705512","article-title":"Stem cell transplantation for the Wiskott-Aldrich syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation","volume":"38","author":"Pai","year":"2006","journal-title":"Bone Marrow Transplant."},{"key":"B12","doi-asserted-by":"publisher","first-page":"1432","DOI":"10.1016\/j.bbmt.2018.03.006","article-title":"A conditioning regimen with plerixafor is safe and improves the outcome of TCRalphabeta(+) and CD19(+) cell-depleted stem cell transplantation in patients with wiskott-aldrich syndrome","volume":"24","author":"Balashov","year":"2018","journal-title":"Biol Blood Marrow Transplant"},{"key":"B13","doi-asserted-by":"publisher","first-page":"1654","DOI":"10.1016\/j.jaci.2018.06.042","article-title":"One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years","volume":"142","author":"Elfeky","year":"2018","journal-title":"J Allergy Clin Immunol"},{"key":"B14","doi-asserted-by":"publisher","first-page":"1428","DOI":"10.1038\/bmt.2012.31","article-title":"Outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome","volume":"47","author":"Shin","year":"2012","journal-title":"Bone Marrow Transplant"},{"key":"B15","doi-asserted-by":"publisher","first-page":"e234","DOI":"10.1097\/MPH.0b013e318279cbfc","article-title":"Impact of conditioning on outcome of hematopoietic stem cell transplantation for wiskott-Aldrich syndrome","volume":"35","author":"Stepensky","year":"2013","journal-title":"J Pediatr Hematol Oncol."},{"key":"B16","first-page":"2175","article-title":"Busulfan\/Fludarabine- or treosulfan\/fludarabine-based conditioning regimen in patients with wiskott-aldrich syndrome given allogeneic hematopoietic cell transplantation \u2014 an EBMT inborn errors working party and scetide retrospective analysis","author":"Albert","year":""},{"key":"B17","doi-asserted-by":"publisher","first-page":"1042","DOI":"10.1016\/j.jaip.2018.07.009","article-title":"Bleeding and splenectomy in Wiskott-Aldrich syndrome: a single-centre experience","volume":"7","author":"Rivers","year":"2018","journal-title":"J Allergy Clin Immunol Pract."},{"key":"B18","doi-asserted-by":"publisher","first-page":"1367","DOI":"10.1182\/blood-2014-09-602573","article-title":"Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome\/X-linked thrombocytopenia","volume":"126","author":"Gerrits","year":"2015","journal-title":"Blood"},{"key":"B19","doi-asserted-by":"publisher","first-page":"619","DOI":"10.1016\/j.jaci.2016.03.007","article-title":"A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation","volume":"138","author":"Brigida","year":"2016","journal-title":"J Allergy Clin Immunol"},{"key":"B20","doi-asserted-by":"publisher","first-page":"e12582","DOI":"10.1111\/dth.12582","article-title":"Successful treatment of pyoderma gangrenosum with anakinra in a patient with Wiskott-Aldrich syndrome","volume":"31","author":"Mercuri","year":"2018","journal-title":"Dermatol Ther"},{"key":"B21","doi-asserted-by":"publisher","first-page":"227ra33","DOI":"10.1126\/scitranslmed.3007280","article-title":"Gene therapy for Wiskott-Aldrich syndrome\u2013long-term efficacy and genotoxicity","volume":"6","author":"Braun","year":"2014","journal-title":"Sci Transl Med."},{"key":"B22","doi-asserted-by":"publisher","first-page":"e239","DOI":"10.1016\/S2352-3026(19)30021-3","article-title":"Lentiviral haemopoietic stem\/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1\/2 clinical study","volume":"6","author":"Ferrua","year":"2019","journal-title":"Lancet Haematol."},{"key":"B23","doi-asserted-by":"publisher","first-page":"1550","DOI":"10.1001\/jama.2015.3253","article-title":"Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome","volume":"313","author":"Hacein-Bey Abina","year":"2015","journal-title":"JAMA"},{"key":"B24","doi-asserted-by":"publisher","first-page":"1327","DOI":"10.1182\/blood-2017-04-777136","article-title":"Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult","volume":"130","author":"Morris","year":"2017","journal-title":"Blood"},{"key":"B25","doi-asserted-by":"publisher","first-page":"825","DOI":"10.1016\/j.jaci.2019.03.012","article-title":"Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome","volume":"144","author":"Sereni","year":"2019","journal-title":"J Allergy Clin Immunol"},{"key":"B26","doi-asserted-by":"publisher","first-page":"668","DOI":"10.1016\/j.jaci.2019.06.039","article-title":"Platelets are the achilles' heel of wiskott-aldrich syndrome","volume":"144","author":"Fischer","year":"2019","journal-title":"J Allergy Clin Immunol"},{"key":"B27","doi-asserted-by":"publisher","first-page":"2046","DOI":"10.1056\/NEJMoa0905506","article-title":"Combined immunodeficiency associated with DOCK8 mutations","volume":"361","author":"Zhang","year":"2009","journal-title":"N Engl J Med."},{"key":"B28","doi-asserted-by":"publisher","first-page":"1289","DOI":"10.1016\/j.jaci.2009.10.038","article-title":"Large deletions and point mutations involving the dedicator of cytokinesis 8 (DOCK8) in the autosomal-recessive form of hyper-IgE syndrome","volume":"124","author":"Engelhardt","year":"2009","journal-title":"J Allergy Clin Immunol"},{"key":"B29","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1007\/s10875-014-0126-0","article-title":"DOCK8 deficiency: clinical and immunological phenotype and treatment options - a review of 136 patients","volume":"35","author":"Aydin","year":"2015","journal-title":"J Clin Immunol."},{"key":"B30","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1111\/imr.12723","article-title":"Insights into immunity from clinical and basic science studies of DOCK8 immunodeficiency syndrome","volume":"287","author":"Su","year":"2019","journal-title":"Immunol Rev."},{"key":"B31","doi-asserted-by":"publisher","first-page":"852","DOI":"10.1016\/j.jaci.2016.02.022","article-title":"Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency","volume":"138","author":"Al-Herz","year":"2016","journal-title":"J Allergy Clin Immunol"},{"key":"B32","doi-asserted-by":"publisher","first-page":"848","DOI":"10.1016\/j.jaip.2018.10.035","article-title":"Hematopoietic stem cell transplantation as treatment for patients with DOCK8 deficiency","volume":"7","author":"Aydin","year":"2018","journal-title":"J Allergy Clin Immunol Pract."},{"key":"B33","doi-asserted-by":"publisher","first-page":"1037","DOI":"10.1016\/j.bbmt.2015.01.022","article-title":"Matched related and unrelated donor hematopoietic stem cell transplantation for DOCK8 deficiency","volume":"21","author":"Cuellar-Rodriguez","year":"2015","journal-title":"Biol Blood Marrow Transplant."},{"key":"B34","doi-asserted-by":"publisher","first-page":"e13015","DOI":"10.1111\/petr.13015","article-title":"Hematopoietic stem cell transplantation from unrelated donors in children with DOCK8 deficiency","volume":"21","author":"Uygun","year":"2017","journal-title":"Pediatr Transplant."},{"key":"B35","doi-asserted-by":"publisher","first-page":"1895","DOI":"10.1016\/j.jaci.2015.11.017","article-title":"Food allergies can persist after myeloablative hematopoietic stem cell transplantation in dedicator of cytokinesis 8-deficient patients","volume":"137","author":"Happel","year":"2016","journal-title":"J Allergy Clin Immunol"},{"key":"B36","doi-asserted-by":"publisher","first-page":"980","DOI":"10.1016\/j.bbmt.2017.03.016","article-title":"Haploidentical related donor hematopoietic stem cell transplantation for dedicator-of-cytokinesis 8 deficiency using post-transplantation cyclophosphamide","volume":"23","author":"Shah","year":"2017","journal-title":"Biol Blood Marrow Transplant."},{"key":"B37","doi-asserted-by":"publisher","first-page":"2144","DOI":"10.1097\/TP.0000000000002649","article-title":"Tandem orthotopic living donor liver transplantation followed by same donor haploidentical hematopoietic stem cell transplantation for DOCK8 deficiency: tandem liver and bone marrow transplant in DOCK8 deficiency","volume":"103","author":"Freeman","year":"2019","journal-title":"Transplantation."},{"key":"B38","doi-asserted-by":"publisher","first-page":"111","DOI":"10.1016\/j.clim.2014.03.006","article-title":"Haploidentical stem cell transplantation in DOCK8 deficiency - Successful control of pre-existing severe viremia with a TCRass\/CD19-depleted graft and antiviral treatment","volume":"152","author":"Ghosh","year":"2014","journal-title":"Clin Immunol"},{"key":"B39","doi-asserted-by":"publisher","first-page":"552","DOI":"10.1038\/bmt.2010.169","article-title":"Curative treatment of autosomal-recessive hyper-IgE syndrome by hematopoietic cell transplantation","volume":"46","author":"Gatz","year":"2011","journal-title":"Bone Marrow Transplant."},{"key":"B40","doi-asserted-by":"publisher","first-page":"339","DOI":"10.1038\/s41409-017-0040-1","article-title":"Successful hematopoietic stem cell transplantation after myeloablative conditioning in three patients with dedicator of cytokinesis 8 deficiency (DOCK8) related Hyper IgE syndrome","volume":"53","author":"Kuskonmaz","year":"2018","journal-title":"Bone Marrow Transplant."},{"key":"B41","doi-asserted-by":"publisher","first-page":"1753","DOI":"10.1016\/j.jaci.2014.03.032","article-title":"Plasmacytoid dendritic cell depletion in DOCK8 deficiency: rescue of severe herpetic infections with IFN-alpha 2b therapy","volume":"133","author":"Keles","year":"2014","journal-title":"J Allergy Clin Immunol"},{"key":"B42","doi-asserted-by":"publisher","first-page":"1456","DOI":"10.1016\/j.jaci.2014.02.008","article-title":"Beneficial IFN-alpha treatment of tumorous herpes simplex blepharoconjunctivitis in dedicator of cytokinesis 8 deficiency","volume":"133","author":"Papan","year":"2014","journal-title":"J Allergy Clin Immunol."},{"key":"B43","doi-asserted-by":"publisher","first-page":"104","DOI":"10.1016\/j.clim.2014.04.005","article-title":"Successful interferon-alpha 2b therapy for unremitting warts in a patient with DOCK8 deficiency","volume":"153","author":"Al-Zahrani","year":"2014","journal-title":"Clin Immunol"},{"key":"B44","doi-asserted-by":"publisher","first-page":"1815","DOI":"10.1038\/s41591-018-0211-7","article-title":"Expanded skin virome in DOCK8-deficient patients","volume":"24","author":"Tirosh","year":"2018","journal-title":"Nat Med"}],"container-title":["Frontiers in Pediatrics"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/article\/10.3389\/fped.2019.00451\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,11,5]],"date-time":"2019-11-05T00:50:46Z","timestamp":1572915046000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/article\/10.3389\/fped.2019.00451\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,11,5]]},"references-count":44,"alternative-id":["10.3389\/fped.2019.00451"],"URL":"https:\/\/doi.org\/10.3389\/fped.2019.00451","relation":{},"ISSN":["2296-2360"],"issn-type":[{"value":"2296-2360","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,11,5]]},"article-number":"451"}}