{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,7]],"date-time":"2026-11-07T02:20:28Z","timestamp":1794018028006,"version":"4.8.0"},"reference-count":61,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2021,4,16]],"date-time":"2021-04-16T00:00:00Z","timestamp":1618531200000},"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. Immunol."],"abstract":"<jats:sec><jats:title>Background<\/jats:title><jats:p>Wiskott Aldrich syndrome (WAS) is characterized by bleeding manifestations, recurrent infections, eczema, autoimmunity, and malignancy. Over the last decade, improved awareness and better in-house diagnostic facilities at several centers in India has resulted in increased recognition of WAS. This study reports collated data across major primary immunodeficiency diseases (PID) centers in India that are involved in care of children with WAS and highlights the varied clinical presentations, genetic profile, and outcomes of patients in India.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Request to share data was sent to multiple centers in India that are involved in care and management of patients with PID. Six centers provided requisite data that were compiled and analyzed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In this multi-institutional cohort, clinical details of 108 patients who had a provisional diagnosis of WAS were received. Of these, 95 patients with \u2018definite WAS\u2019 were included Fourteen patients were classified as XLT and 81 patients as WAS. Median age at onset of symptoms of patients was 3 months (IQR 1.6, 6.0 months) and median age at diagnosis was 12 months (IQR 6,48 months). Clinical profile included bleeding episodes (92.6%), infections (84.2%), eczema (78.9%), various autoimmune manifestations (40%), and malignancy (2.1%). DNA analysis revealed 47 variants in 67 cases. Nonsense and missense variants were the most common (28.4% each), followed by small deletions (19.4%), and splice site defects (16.4%). We also report 24 novel variants, most of these being frameshift and nonsense mutations resulting in premature termination of protein synthesis. Prophylactic intravenous immunoglobulin (IVIg) was initiated in 52 patients (54.7%). Hematopoietic stem cell transplantation (HSCT) was carried out in 25 patients (26.3%). Of those transplanted, disease-free survival was seen in 15 patients (60%). Transplant related mortality was 36%. Outcome details were available for 89 patients. Of these, 37% had died till the time of this analysis. Median duration of follow-up was 36 months (range 2 weeks- 12 years; IQR 16.2 months- 70 months).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>We report the first nationwide cohort of patients with WAS from India. Bleeding episodes and infections are common manifestations. Mortality continues to be high as curative therapy is not accessible to most of our patients.<\/jats:p><\/jats:sec>","DOI":"10.3389\/fimmu.2021.627651","type":"journal-article","created":{"date-parts":[[2021,4,16]],"date-time":"2021-04-16T17:14:12Z","timestamp":1618593252000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":43,"title":["Wiskott Aldrich Syndrome: A Multi-Institutional Experience From India"],"prefix":"10.3389","volume":"12","author":[{"given":"Deepti","family":"Suri","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rashmi","family":"Rikhi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ankur K.","family":"Jindal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amit","family":"Rawat","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Murugan","family":"Sudhakar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pandiarajan","family":"Vignesh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anju","family":"Gupta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anit","family":"Kaur","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jyoti","family":"Sharma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jasmina","family":"Ahluwalia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Prateek","family":"Bhatia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alka","family":"Khadwal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Revathi","family":"Raj","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ramya","family":"Uppuluri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mukesh","family":"Desai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Prasad","family":"Taur","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ambreen A.","family":"Pandrowala","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vijaya","family":"Gowri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manisha R.","family":"Madkaikar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harsha Prasada","family":"Lashkari","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sagar","family":"Bhattad","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Harish","family":"Kumar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sanjeev","family":"Verma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kohsuke","family":"Imai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shigeaki","family":"Nonoyama","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Osamu","family":"Ohara","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Koon W.","family":"Chan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pamela P.","family":"Lee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yu Lung","family":"Lau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Surjit","family":"Singh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2021,4,16]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1007\/BF00916954","article-title":"Primary immunodeficiencies in Switzerland: first report of the national registry in adults and children","volume":"8","author":"Ryser","year":"1988","journal-title":"J Clin Immunol"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1016\/s0022-3476(80)80133-8","article-title":"The Wiskott-Aldrich syndrome in the United States and Canada (1892-1979)","volume":"97","author":"Perry","year":"1980","journal-title":"J Pediatr"},{"key":"B3","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2006.02.005","article-title":"The Wiskott-Aldrich syndrome","volume":"117","author":"Ochs","year":"2006","journal-title":"J Allergy Clin Immunol"},{"key":"B4","doi-asserted-by":"publisher","first-page":"84","DOI":"10.1016\/j.bbmt.2008.10.007","article-title":"Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment","volume":"15","author":"Ochs","year":"2009","journal-title":"Biol Blood Marrow Transplant"},{"key":"B5","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1016\/0888-7543(91)90480-3","article-title":"Localization of the gene for the Wiskott-Aldrich syndrome between two flanking markers, TIMP and DXS255, on Xp11.22-Xp11.3","volume":"10","author":"Kwan","year":"1991","journal-title":"Genomics"},{"key":"B6","doi-asserted-by":"publisher","DOI":"10.1128\/mcb.15.10.5725","article-title":"Wiskott-Aldrich syndrome protein physically associates with Nck through Src homology 3 domains","volume":"15","author":"Rivero-Lezcano","year":"1995","journal-title":"Mol Cell Biol"},{"key":"B7","doi-asserted-by":"publisher","DOI":"10.1016\/s0092-8674(00)81050-8","article-title":"Wiskott-Aldrich syndrome protein, a novel effector for the GTPase CDC42Hs, is implicated in actin polymerization","volume":"84","author":"Symons","year":"1996","journal-title":"Cell"},{"key":"B8","first-page":"195","article-title":"Tyrosine kinase signaling regulates Wiskott-Aldrich syndrome protein function, which is essential for megakaryocyte differentiation","volume":"8","author":"Miki","year":"1997","journal-title":"Cell Growth Differ"},{"key":"B9","doi-asserted-by":"publisher","DOI":"10.1006\/bbrc.1997.8064","article-title":"Direct binding of the verprolin-homology domain in N-WASP to actin is essential for cytoskeletal reorganization","volume":"243","author":"Miki","year":"1998","journal-title":"Biochem Biophys Res Commun"},{"key":"B10","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V90.1.70.70_70_75","article-title":"Expression of Wiskott-Aldrich syndrome protein (WASP) gene during hematopoietic differentiation","author":"Parolini","year":"1997","journal-title":"Blood"},{"key":"B11","doi-asserted-by":"crossref","DOI":"10.4049\/jimmunol.157.9.3791","article-title":"Evidence that the Wiskott-Aldrich syndrome protein may be involved in lymphoid cell signaling pathways","volume":"157","author":"Cory","year":"1996","journal-title":"J Immunol"},{"key":"B12","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V90.8.3089","article-title":"Defective actin reorganization and polymerization of Wiskott-Aldrich T cells in response to CD3-mediated stimulation","volume":"90","author":"Gallego","year":"1997","journal-title":"Blood"},{"key":"B13","doi-asserted-by":"publisher","DOI":"10.1016\/s1074-7613(00)00046-7","article-title":"Cbl-b is a negative regulator of receptor clustering and raft aggregation in T cells","volume":"13","author":"Krawczyk","year":"2000","journal-title":"Immunity"},{"key":"B14","doi-asserted-by":"publisher","DOI":"10.1016\/s1097-2765(02)00728-1","article-title":"Mechanism of recruitment of WASP to the immunological synapse and of its activation following TCR ligation","volume":"10","author":"Sasahara","year":"2002","journal-title":"Mol Cell"},{"key":"B15","doi-asserted-by":"publisher","DOI":"10.1016\/s1074-7613(02)00360-6","article-title":"Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation","volume":"17","author":"Dupr\u00e9","year":"2002","journal-title":"Immunity"},{"key":"B16","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.162376099","article-title":"Wiskott-Aldrich syndrome protein is required for NK cell cytotoxicity and colocalizes with actin to NK cell-activating immunologic synapses","volume":"99","author":"Orange","year":"2002","journal-title":"Proc Natl Acad Sci U.S.A."},{"key":"B17","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2009-08-237560","article-title":"Systemic autoimmunity and defective Fas ligand secretion in the absence of the Wiskott-Aldrich syndrome protein","volume":"116","author":"Nikolov","year":"2010","journal-title":"Blood"},{"key":"B18","doi-asserted-by":"publisher","DOI":"10.1084\/jem.190.9.1329","article-title":"Antigen receptor-induced activation and cytoskeletal rearrangement are impaired in Wiskott-Aldrich syndrome protein-deficient lymphocytes","volume":"190","author":"Zhang","year":"1999","journal-title":"J Exp Med"},{"key":"B19","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V95.9.2943.009k17_2943_2946","article-title":"Wiskott-Aldrich syndrome protein is necessary for efficient IgG-mediated phagocytosis","volume":"95","author":"Lorenzi","year":"2000","journal-title":"Blood"},{"key":"B20","doi-asserted-by":"publisher","DOI":"10.4049\/jimmunol.165.1.221","article-title":"The polarization defect of Wiskott-Aldrich syndrome macrophages is linked to dislocalization of the Arp2\/3 complex","volume":"165","author":"Linder","year":"2000","journal-title":"J Immunol"},{"key":"B21","doi-asserted-by":"publisher","DOI":"10.1016\/s1357-2725(01)00162-5","article-title":"Restoration of podosomes and chemotaxis in Wiskott-Aldrich syndrome macrophages following induced expression of WASp","volume":"34","author":"Jones","year":"2002","journal-title":"Int J Biochem Cell Biol"},{"key":"B22","doi-asserted-by":"crossref","DOI":"10.4049\/jimmunol.161.2.1026","article-title":"Monocytes from Wiskott-Aldrich patients display reduced chemotaxis and lack of cell polarization in response to monocyte chemoattractant protein-1 and formyl-methionyl-leucyl-phenylalanine","volume":"161","author":"Badolato","year":"1998","journal-title":"J Immunol"},{"key":"B23","article-title":"Clinical profile and genetic basis of Wiskott-Aldrich syndrome at Chandigarh, North India","volume":"30","author":"Suri","year":"2012","journal-title":"Asian Pac J Allergy Immunol"},{"key":"B24","doi-asserted-by":"publisher","DOI":"10.1111\/j.1600-0609.2012.01818.x.Epub2012Jul14","article-title":"Molecular basis of Wiskott-Aldrich syndrome in patients from India","volume":"89","author":"David","year":"2012","journal-title":"Eur J Haematol"},{"key":"B25","doi-asserted-by":"publisher","DOI":"10.1007\/s13312-018-1354-9","article-title":"Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency Disorders: Experience from a Referral Center in India","volume":"55","author":"Uppuluri","year":"2018","journal-title":"Indian Pediatr"},{"key":"B26","doi-asserted-by":"publisher","DOI":"10.1007\/s10875-019-00600-z","article-title":"Haploidentical Stem Cell Transplantation with Post-Transplant Cyclophosphamide for Primary Immune Deficiency Disorders in Children: Challenges and Outcome from a Tertiary Care Center in South India","volume":"39","author":"Uppuluri","year":"2019","journal-title":"J Clin Immunol"},{"key":"B27","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.26196","article-title":"Sclerosing cholangitis and intracranial lymphoma in a child with classical Wiskott-Aldrich syndrome","volume":"64","author":"Vignesh","year":"2017","journal-title":"Pediatr Blood Cancer"},{"key":"B28","doi-asserted-by":"publisher","first-page":"7","DOI":"10.1007\/s10875-018-0585-9","article-title":"Monoclonal Gammopathy of Unclear Significance in a Child with Wiskott-Aldrich Syndrome: a Rare Occurrence","volume":"39","author":"Rikhi","year":"2019","journal-title":"J Clin Immunol"},{"key":"B29","doi-asserted-by":"publisher","DOI":"10.1007\/s13312-017-1097-z","article-title":"Un-manipulated Haploidentical Transplant in Wiskott-Aldrich Syndrome","volume":"54","author":"John","year":"2017","journal-title":"Indian Pediatr"},{"key":"B30","article-title":"Wiskott-Aldrich Syndrome","volume":"12","author":"Gupta","year":"1964","journal-title":"A Case Rep J Assoc Physicians India"},{"key":"B31","article-title":"Successful bone marrow transplantation in an infant with Wiskott-Aldrich syndrome","volume":"36","author":"Mathew","year":"1999","journal-title":"Indian Pediatr"},{"key":"B32","doi-asserted-by":"publisher","DOI":"10.1007\/BF02730830","article-title":"Management of Wiskott-Aldrich syndrome","volume":"63","author":"Srivastava","year":"1996","journal-title":"Indian J Pediatr"},{"key":"B33","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2017.00714","article-title":"Primary Immunodeficiency Disorders in India-A Situational Review","volume":"8","author":"Jindal","year":"2017","journal-title":"Front Immunol"},{"key":"B34","doi-asserted-by":"publisher","first-page":"327","DOI":"10.1007\/s13312-017-1097-z","article-title":"Un-manipulated Haploidentical Transplant in Wiskott-Aldrich Syndrome","volume":"54","author":"John","year":"2017","journal-title":"Indian Pediatr"},{"key":"B35","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V90.7.2680.2680_2680_2689","article-title":"Wiskott-Aldrich syndrome\/X-linked thrombocytopenia: WASP gene mutations, protein expression, and phenotype","volume":"90","author":"Zhu","year":"1997","journal-title":"Blood"},{"key":"B36","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaci.2003.07.003","article-title":"Pediatric AIDS Clinical Trials GroupLymphocyte subsets in healthy children from birth through 18 years of age: the Pediatric AIDS Clinical Trials Group P1009 study","volume":"112","author":"Shearer","year":"2003","journal-title":"J Allergy Clin Immunol"},{"key":"B37","doi-asserted-by":"publisher","DOI":"10.4274\/tjh.galenos.2020.2020.0334","article-title":"Clinical Features and Outcomes of 23 Patients with Wiskott-Aldrich Syndrome: A Single-Center Experience","volume":"37","author":"Haskolo\u011flu","year":"2020","journal-title":"Turk J Haematol"},{"key":"B38","doi-asserted-by":"publisher","DOI":"10.1016\/s0022-3476(05)82002-5","article-title":"A multiinstitutional survey of the Wiskott-Aldrich syndrome","volume":"125","author":"Sullivan","year":"1994","journal-title":"J Pediatr"},{"key":"B39","doi-asserted-by":"publisher","first-page":"490","DOI":"10.1007\/s10875-009-9285-9","article-title":"Clinical and molecular characteristics of 35 Chinese children with Wiskott-Aldrich syndrome","volume":"29","author":"Lee","year":"2009","journal-title":"J Clin Immunol"},{"key":"B40","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2019.01549","article-title":"When WAS Gene Diagnosis Is Needed: Seeking Clues Through Comparison Between Patients with Wiskott-Aldrich Syndrome and Idiopathic Thrombocytopenic Purpura","volume":"10","author":"Jin","year":"2019","journal-title":"Front Immunol"},{"key":"B41","doi-asserted-by":"publisher","DOI":"10.1177\/2050313X17753788","article-title":"Wiskott-Aldrich syndrome that was initially diagnosed as immune thrombocytopenic purpura secondary to a cytomegalovirus infection","volume":"6","author":"Kaneko","year":"2018","journal-title":"SAGE Open Med Case Rep"},{"key":"B42","article-title":"Postnatal cytomegalovirus infection in an infant with congenital thrombocytopenia: how it can support or mislead the diagnosis of Wiskott-Aldrich syndrome","volume":"24","author":"Poddighe","year":"2016","journal-title":"Infez Med"},{"key":"B43","doi-asserted-by":"publisher","DOI":"10.1016\/j.jaapos.2013.08.007","article-title":"Unilateral recalcitrant glaucoma associated with cytomegalovirus in an immunocompromised child with Wiskott-Aldrich syndrome","volume":"17","author":"Kaushik","year":"2013","journal-title":"J AAPOS"},{"key":"B44","doi-asserted-by":"publisher","DOI":"10.1097\/00130832-200112000-00003","article-title":"Mycobacterial diseases in primary immunodeficiencies","volume":"1","author":"Reichenbach","year":"2001","journal-title":"Curr Opin Allergy Clin Immunol"},{"key":"B45","doi-asserted-by":"publisher","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":"B46","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-05-1480","article-title":"Clinical course of patients with WASP gene mutations","volume":"103","author":"Imai","year":"2004","journal-title":"Blood"},{"key":"B47","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019002939","article-title":"Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report","volume":"135","author":"Shin","year":"2020","journal-title":"Blood"},{"key":"B48","doi-asserted-by":"publisher","DOI":"10.1542\/peds.111.5.e622","article-title":"Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients","volume":"111","author":"Dupuis-Girod","year":"2003","journal-title":"Pediatrics"},{"key":"B49","doi-asserted-by":"publisher","DOI":"10.1016\/s0022-3476(96)70248-2","article-title":"Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center","volume":"129","author":"Ozsahin","year":"1996","journal-title":"J Pediatr"},{"key":"B50","doi-asserted-by":"publisher","DOI":"10.1111\/j.1440-1754.1992.tb02703.x","article-title":"Takayasu\u2019s arteritis associated with Wiskott-Aldrich syndrome","volume":"28","author":"Lau","year":"1992","journal-title":"J Paediatr Child Health"},{"key":"B51","doi-asserted-by":"publisher","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":"B52","doi-asserted-by":"publisher","first-page":"333","DOI":"10.1016\/j.jaci.2018.04.017","article-title":"Screening for Wiskott-Aldrich syndrome by flow cytometry","volume":"142","author":"Chiang","year":"2018","journal-title":"J Allergy Clin Immunol"},{"key":"B53","doi-asserted-by":"publisher","DOI":"10.1016\/j.molimm.2010.11.013","article-title":"Genetic characteristics of eighty-seven patients with the Wiskott-Aldrich syndrome","volume":"48","author":"Gul\u00e1csy","year":"2011","journal-title":"Mol Immunol"},{"key":"B54","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2003-05-1592","article-title":"Mutations of the Wiskott-Aldrich Syndrome Protein (WASP): hotspots, effect on transcription, and translation and phenotype\/genotype correlation","volume":"104","author":"Jin","year":"2004","journal-title":"Blood"},{"key":"B55","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019002939","article-title":"Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report","volume":"135","author":"Burroughs","year":"2020","journal-title":"Blood"},{"key":"B56","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2016.158808","article-title":"A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome","volume":"102","author":"Shekhovtsova","year":"2017","journal-title":"Haematologica"},{"key":"B57","doi-asserted-by":"publisher","first-page":"593","DOI":"10.1007\/s10875-010-9381-x","article-title":"Clinical aspects and genetic analysis of taiwanese patients with wiskott-Aldrich syndrome protein mutation: the first identification of x-linked thrombocytopenia in the chinese with novel mutations","volume":"30","author":"Lee","year":"2010","journal-title":"J Clin Immunol"},{"key":"B58","doi-asserted-by":"publisher","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":"B59","doi-asserted-by":"publisher","first-page":"15","DOI":"10.1007\/s10875-014-0105-5","article-title":"Hematopoietic Stem Cell Transplantation for X-Linked Thrombocytopenia with Mutations in the WAS gene","volume":"35","author":"Oshima","year":"2015","journal-title":"J Clin Immunol"},{"key":"B60","doi-asserted-by":"publisher","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":"B61","doi-asserted-by":"publisher","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"}],"container-title":["Frontiers in Immunology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fimmu.2021.627651\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,2]],"date-time":"2023-11-02T06:41:02Z","timestamp":1698907262000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fimmu.2021.627651\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,4,16]]},"references-count":61,"alternative-id":["10.3389\/fimmu.2021.627651"],"URL":"https:\/\/doi.org\/10.3389\/fimmu.2021.627651","relation":{},"ISSN":["1664-3224"],"issn-type":[{"value":"1664-3224","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,4,16]]},"article-number":"627651"}}