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Current and emerging treatment options for Wiskott–Aldrich syndrome

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Read on this site (5)

Jia Luo, Jie Peng, Pei-Quan Zhao & Ping Fei. (2023) CMV Retinitis in Wiskott Aldrich Syndrome. Ocular Immunology and Inflammation 31:1, pages 134-141.
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Smitha Hosahalli Vasanna, Maria A Pereda & Jignesh Dalal. (2021) Clinical Features, Cancer Biology, Transplant Approach and Other Integrated Management Strategies for Wiskott–Aldrich Syndrome. Journal of Multidisciplinary Healthcare 14, pages 3497-3512.
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Marton Keszei, Joanna S. Kritikou, Deborah Sandfort, Minghui He, Mariana M.S. Oliveira, Hannah Wurzer, Raoul V. Kuiper & Lisa S. Westerberg. (2018) Wiskott-Aldrich syndrome gene mutations modulate cancer susceptibility in the p53± murine model. OncoImmunology 7:9.
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Jose Maria Bastida, Monica Del Rey, Nuria Revilla, Rocio Benito, Martin Perez-Andrés, Berta González, Susana Riesco, Kamila Janusz, Jose Padilla, Ana Hortal Benito-Sendin, David Bueno, Elena Blanco, Maria Hernández-Rivas, Vicente Vicente, Jose Rivera, Ramon González-Porras & Maria Luisa Lozano. (2017) Wiskott–Aldrich syndrome in a child presenting with macrothrombocytopenia. Platelets 28:4, pages 417-420.
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Claire Booth, Juliana Silva & Paul Veys. (2016) Stem cell transplantation for the treatment of immunodeficiency in children: current status and hopes for the future. Expert Review of Clinical Immunology 12:7, pages 713-723.
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Articles from other publishers (48)

Fangfang Liang, Cheng Peng, Xianze Luo, Linlin Wang, Yanyan Huang, Le Yin, Luming Yue, Jun Yang & Xiaodong Zhao. (2023) A single-cell atlas of immunocytes in the spleen of a mouse model of Wiskott-Aldrich syndrome. Cellular Immunology 393-394, pages 104783.
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Melissa Pille, John Avila, Guillem Sanchez Sanchez, Glenn Goetgeluk, Stijn De Munter, Hanne Jansen, Lore Billiet, Karin Weening, Haipeng Xue, Sarah Bonte, Joline Ingels, Laurenz De Cock, Eva Pascal, Lucas Deseins, Tessa Kerre, Tom Taghon, Georges Leclercq, David Vermijlen, Brian Davis & Bart Vandekerckhove. (2023) The Wiskott–Aldrich syndrome protein is required for positive selection during T-cell lineage differentiation. Frontiers in Immunology 14.
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Smitha Hosahalli Vasanna & Jignesh Dalal. (2023) Traffic jam within lymphocytes: A clinician’s perspective. Frontiers in Immunology 13.
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Hou-Yuan Qiu, Rui-Jin Ji & Ying Zhang. (2022) Current advances of CRISPR-Cas technology in cell therapy. Cell Insight 1:6, pages 100067.
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Qingzhou Meng, Haixiang Sun & Jianghuai Liu. (2022) Precise somatic genome editing for treatment of inborn errors of immunity. Frontiers in Immunology 13.
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Asma Naseem, Zohar Steinberg & Alessia Cavazza. (2022) Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome. Frontiers in Immunology 13.
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Allison M. Lloyd-McLennan, Kristin A. Shimano, Morna Dorsey & Kelly M. Cordoro. (2022) Eczema and Petechiae in an Infant. Pediatrics In Review 43:7, pages e19-e23.
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Alkistis Adramerina, Nancy Chainoglou, Stamatia Theodoridou, Aikaterini Teli & Marina Economou. (2022) Off Label Use of Eltrombopag and Recombinant Activated Factor VII in Wiskott-Aldrich Syndrome. A Case Report and Review of Literature. Clinical Pediatric Hematology-Oncology 29:1, pages 12-16.
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Malek Benakli, Redhouane Ahmed Nacer, Farih Mehdid, Nadia Rahmoune, Mounira Baazizi, Dina Ait Ouali, Hanane Bouarab, Sara Zerkout, Farida Harieche & Rose-Marie Hamladji. (2022) Successful Allogeneic Peripheral Blood Stem Cell Transplantation in 4 Wiskott-Aldrich Syndrome Patients. Journal of Pediatric Hematology/Oncology 44:2, pages e324-e328.
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Qin Zhao, Rongxin Dai, Yanan Li, Yanping Wang, Xuemei Chen, Zhou Shu, Lina Zhou, Yuan Ding, Xuemei Tang & Xiaodong Zhao. (2021) Trends in TREC values according to age and gender in Chinese children and their clinical applications. European Journal of Pediatrics 181:2, pages 529-538.
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Jenny Huang & Pavadee Poowuttikul. 2022. Absolute Allergy and Immunology Board Review. Absolute Allergy and Immunology Board Review 235 249 .
Akash Kumar, Saurav Jain, Prawin Kumar & Jagdish Prasad Goyal. (2021) Generalised eczema: a diagnostic clue to Wiskott-Aldrich syndrome. BMJ Case Reports 14:6, pages e242642.
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Elizabeth Rivers, Ying Hong, Mona Bajaj‐Elliott, Austen Worth & Adrian J. Thrasher. (2021) IL‐18: A potential inflammation biomarker in Wiskott–Aldrich syndrome. European Journal of Immunology 51:5, pages 1285-1288.
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Elisa Ochfeld, Dannielle Grayer, Ruchika Sharma, Jennifer Schneiderman, Lisa Giordano & Melanie Makhija. (2021) A Novel Mutation in WAS Gene Causing a Phenotypic Presentation of Wiskott-Aldrich Syndrome: A Case Report. Journal of Pediatric Hematology/Oncology 43:2, pages e234-e236.
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Tianping Chen, Jun Sun, Guanghui Liu, Chuangao Yin, Haipeng Liu, Lijun Qu, Shijin Fang, Ash Shifra & Gil Gilad. (2021) A Homozygous Mutation in 5′ Untranslated Region of TNFRSF11A Leading to Molecular Diagnosis of Osteopetrosis Coinheritance With Wiskott-Aldrich Syndrome. Journal of Pediatric Hematology/Oncology 43:2, pages e264-e267.
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Juanjuan Zhou, Quanmei Yan, Chengcheng Tang, Yuan Liao, Quanjun Zhang, Xiaomin Wang, Xiaoqing Zhou, Liangxue Lai & Qingjian Zou. (2020) Development of a rabbit model of Wiskott‐Aldrich syndrome. The FASEB Journal 35:2.
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Megan A. Cooper, Ofer Zimmerman, Ramya Nataraj & Robert F. Wynn. (2021) Lifelong Immune Modulation Versus Hematopoietic Cell Therapy for Inborn Errors of Immunity. The Journal of Allergy and Clinical Immunology: In Practice 9:2, pages 628-639.
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SunilVishan Kapur & JitendraS Oswal. (2021) Atypical presentation of Wiskott–Aldrich syndrome with autoimmune arthritis and macrothrombocytopenia. Indian Journal of Rheumatology 16:4, pages 469.
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Rajeev RaiAdrian J. ThrasherAlessia Cavazza. (2021) Gene Editing for the Treatment of Primary Immunodeficiency Diseases. Human Gene Therapy 32:1-2, pages 43-51.
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Jérôme Mastio, Mezida B. Saeed, Hannah Wurzer, Max Krecke, Lisa S. Westerberg & Clément Thomas. (2020) Higher Incidence of B Cell Malignancies in Primary Immunodeficiencies: A Combination of Intrinsic Genomic Instability and Exocytosis Defects at the Immunological Synapse. Frontiers in Immunology 11.
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Elizabeth Rivers, Rajeev Rai, Jonas Lötscher, Michael Hollinshead, Gasper Markelj, James Thaventhiran, Austen Worth, Alessia Cavazza, Christoph Hess, Mona Bajaj-Elliott & Adrian J Thrasher. (2020) Wiskott Aldrich syndrome protein regulates non-selective autophagy and mitochondrial homeostasis in human myeloid cells. eLife 9.
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Rajeev Rai, Marianna Romito, Elizabeth Rivers, Giandomenico Turchiano, Georges Blattner, Winston Vetharoy, Dariusz Ladon, Geoffroy Andrieux, Fang Zhang, Marta Zinicola, Diego Leon-Rico, Giorgia Santilli, Adrian J. Thrasher & Alessia Cavazza. (2020) Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome. Nature Communications 11:1.
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Ali Abbara, Sophie A. Clarke, Rosalind Brewster, Alexia Simonnard, Pei Chia Eng, Maria Phylactou, Deborah Papadopoulou, Chioma Izzi-Engbeaya, Amir H. Sam, Florian Wernig, Eliza Jonauskyte, Alexander N. Comninos, Karim Meeran, Tom W. Kelsey & Waljit S. Dhillo. (2020) Pharmacodynamic Response to Anti-thyroid Drugs in Graves' Hyperthyroidism. Frontiers in Endocrinology 11.
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O. A. Shvets, D. S. Abramov, A. L. Khoreva, D. E. Pershin, A. M. Kieva & A. V. Phonkin. (2020) Rosay–Dorfman – like lymphadenopathy in a patient with Wiskott–Aldrich syndrome: diagnostic difficulties. Pediatric Hematology/Oncology and Immunopathology 19:1, pages 108-115.
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Julie V. Schaffer, Melanie Makhija & Amy S. Paller. 2019. Harper's Textbook of Pediatric Dermatology. Harper's Textbook of Pediatric Dermatology 2028 2066 .
Ying-Ying Jin, Jing Wu, Tong-Xin Chen & Ji Chen. (2019) When WAS Gene Diagnosis Is Needed: Seeking Clues Through Comparison Between Patients With Wiskott-Aldrich Syndrome and Idiopathic Thrombocytopenic Purpura. Frontiers in Immunology 10.
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E. B. Machneva, E. V. Skorobogatova, E. A. Pristanskova, V. V. Konstantinova, A. E. Burya, L. V. Olkhova, N. V. Sidorova, A. V. Mezentseva, K. I. Kirgizov, O. L. Blagonravova, Yu. A. Nikolaeva, O. A. Filina, S. C. Vakhlyarskaya, Yu. V. Skvortsova, I. V. Kondratenko, A. A. Bologov & A. A. Maschan. (2019) The experience of hematopoietic stem cell transplantation in primary immunodeficiencies in the Russian Children's Clinical Hospital. Pediatric Hematology/Oncology and Immunopathology 18:2, pages 30-42.
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Richard Mitchell. (2019) Hematopoietic Stem Cell Transplantation Beyond Severe Combined Immunodeficiency: Seeking a Cure for Primary Immunodeficiency. The Journal of Allergy and Clinical Immunology: In Practice 7:3, pages 776-785.
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Meng-Meng Duan & Hua-Jun Zheng. (2019) Carrier rate analysis of single-gene disorders based on 1000 genome project and exome aggregation consortium data. Reproductive and Developmental Medicine 3:4, pages 235.
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David T Ewart, Erik J Peterson & Clifford J Steer. (2019) Gene editing for inflammatory disorders. Annals of the Rheumatic Diseases 78:1, pages 6-15.
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Rakesh Kumar Pilania, Rashmi Rekhi & Deepti Suri. 2019. Pediatric Immunology. Pediatric Immunology 701 704 .
Yiping Fan & Jerry Kok Yen Chan. (2018) Editing the Genome Ex Vivo Stem Cell Therapy. Current Stem Cell Reports 4:4, pages 338-345.
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Yan Yue, Xiaodong Shi, Zeliang Song, Jiayue Qin, Junhui Li, Shunqiao Feng & Rong Liu. (2018) Posttransplant cyclophosphamide for haploidentical stem cell transplantation in children with Wiskott-Aldrich syndrome. Pediatric Blood & Cancer 65:8, pages e27092.
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Michael P. Rettig. (2018) Get Outta Here! Addition of Mobilizing Agents to Conditioning Regimen Improves Donor Engraftment after Allogeneic Hematopoietic Stem Cell Transplantation for Wiskott-Aldrich Syndrome. Biology of Blood and Marrow Transplantation 24:7, pages 1309-1311.
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Maria A. Karalexi, Marianna Tzanoudaki, Andreas Fryganas, Alexia Gkergki, Dora Spyropoulou, Anna Papadopoulou, Vassiliki Papaevangelou & Ioannis Petrocheilos. (2018) Wiskott-Aldrich Syndrome Misdiagnosed as Immune Thrombocytopenic Purpura: A Case Report. Journal of Pediatric Hematology/Oncology 40:3, pages 240-242.
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Andrea M. Byrne, Tal Schechter & Robyn Westmacott. (2018) Neuropsychological Profile of a Girl with Wiskott-Aldrich Syndrome. Cognitive and Behavioral Neurology 31:1, pages 13-17.
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Alejandra Gutierrez-Guerrero, Sabina Sanchez-Hernandez, Giuseppe Galvani, Javier Pinedo-Gomez, Rocio Martin-Guerra, Almudena Sanchez-Gilabert, Araceli Aguilar-González, Marién Cobo, Philip GregoryMichael HolmesKarim Benabdellah & Francisco Martin. (2018) Comparison of Zinc Finger Nucleases Versus CRISPR-Specific Nucleases for Genome Editing of the Wiskott-Aldrich Syndrome Locus. Human Gene Therapy 29:3, pages 366-380.
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M. Ettinger, J. Schreml, K. Wirsching, M. Berneburg & S. Schreml. (2018) Skin signs of primary immunodeficiencies: how to find the genes to check. British Journal of Dermatology 178:2, pages 335-349.
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Juliana Silva, Claire Booth & Paul Veys. 2018. Management of Infections in the Immunocompromised Host. Management of Infections in the Immunocompromised Host 375 393 .
Alessandro Plebani, Gaetano Chirico & Vassilios Lougaris. 2018. Neonatology. Neonatology 1607 1618 .
Elizabeth Rivers & Adrian J. Thrasher. (2017) Wiskott-Aldrich syndrome protein: Emerging mechanisms in immunity. European Journal of Immunology 47:11, pages 1857-1866.
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Emma C. Morris, Thomas FoxRonjon ChakravertyRita TendeiroKatie SnellChristine RivatSarah GraceKimberly GilmourSarita Workman, Karen BucklandKatie ButlerRonnie Chee, Alan D. SalamaHazem IbrahimHavinder HaraCecile DuretFulvio Mavilio, Frances Male, Frederick D. Bushman, Anne Galy, Siobhan O. Burns, H. Bobby GasparAdrian J. Thrasher. (2017) Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. Blood 130:11, pages 1327-1335.
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Salah Ali, Anna Gacsadi, Elizabeth McDougall, Christine Armstrong, Joerg Krueger, Tal Schechter & Muhammad Ali. (2017) Successful Reduced Intensity Allogeneic Transplant With Full Donor Chimerism and Good Quality of Life in Adolescent Patient With Wiskott-Aldrich Syndrome. Journal of Pediatric Hematology/Oncology 39:5, pages e267-e269.
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Adrian J. Thrasher & David A. Williams. (2017) Evolving Gene Therapy in Primary Immunodeficiency. Molecular Therapy 25:5, pages 1132-1141.
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Andrew R. Gennery, Laszlo Marodi, John B. Ziegler, Teresa Español & Bodo Grimbacher. 2017. Primary Immunodeficiency Diseases. Primary Immunodeficiency Diseases 461 517 .
Alessandro Plebani, Gaetano Chirico & Vassilios Lougaris. 2019. Neonatology. Neonatology 1 11 .
Linda M. Griffith, Morton J. Cowan, Luigi D. Notarangelo, Donald B. Kohn, Jennifer M. Puck, William T. Shearer, Lauri M. Burroughs, Troy R. Torgerson, Hélène Decaluwe & Elie Haddad. (2016) Primary Immune Deficiency Treatment Consortium (PIDTC) update. Journal of Allergy and Clinical Immunology 138:2, pages 375-385.
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Alessandro Plebani, Gaetano Chirico & Vassilios Lougaris. 2016. Neonatology. Neonatology 1 11 .

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