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Kanwaldeep K Mallhi, Aleksandra Petrovic & Hans D Ochs. (2021) Hematopoietic Stem Cell Therapy for Wiskott–Aldrich Syndrome: Improved Outcome and Quality of Life. Journal of Blood Medicine 12, pages 435-447.
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Fabien Touzot, Salima Hacein-Bey-Abina, Alain Fischer & Marina Cavazzana. (2014) Gene therapy for inherited immunodeficiency. Expert Opinion on Biological Therapy 14:6, pages 789-798.
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Shulian Zhang, Rong Zhang, Chao Chen & Jinqiao Sun. (2013) Two cases of wiskott–Aldrich syndrome in neonates due to gene mutations. Fetal and Pediatric Pathology 32:4, pages 312-315.
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Giorgia Santilli, Susannah I Thornhill, Christine Kinnon & Adrian J Thrasher. (2008) Gene therapy of inherited immunodeficiencies. Expert Opinion on Biological Therapy 8:4, pages 397-407.
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Articles from other publishers (36)
Anne Galy & Marie Dewannieux. (2023) Recent advances in hematopoietic gene therapy for genetic disorders. Archives de Pédiatrie 30:8, pages 8S24-8S31.
Crossref
Crossref
Xin Ji, Xuening Hou, Xin Guo, Yifeng Sun, Futian Ma & Jihong Hao. (2022) Identification of a novel WAS mutation and the non-splicing effect of a second-site mutation in a Chinese pedigree with Wiskott–Aldrich syndrome. Orphanet Journal of Rare Diseases 17:1.
Crossref
Crossref
Benjamin C. Houghton & Claire Booth. (2021) Gene Therapy for Primary Immunodeficiency. HemaSphere 5:1, pages e509.
Crossref
Crossref
Laura De Rosa, Maria Carmela Latella, Alessia Secone Seconetti, Cecilia Cattelani, Johann W. Bauer, Sergio Bondanza & Michele De Luca. (2020) Toward Combined Cell and Gene Therapy for Genodermatoses. Cold Spring Harbor Perspectives in Biology 12:5, pages a035667.
Crossref
Crossref
Maria Pia Cicalese & Alessandro Aiuti. 2018. Management of Infections in the Immunocompromised Host. Management of Infections in the Immunocompromised Host
413
431
.
Maximilian Müller, Tafadzwa Mlambo & Claudio Mussolino. 2017. Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders. Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders
137
157
.
Alessandra Magnani & Nizar Mahlaoui. (2016) Managing Inflammatory Manifestations in Patients with Chronic Granulomatous Disease. Pediatric Drugs 18:5, pages 335-345.
Crossref
Crossref
Jing-Wen Xie, Zhi-Yong Zhang, Jun-Feng Wu, Da-Wei Liu, Wei Liu, Yao Zhao, Li-Ping Jiang, Xue-Mei Tang, Mo Wang & Xiao-Dong Zhao. (2015) In vivo reversion of an inherited mutation in a Chinese patient with Wiskott–Aldrich syndrome. Human Immunology 76:6, pages 406-413.
Crossref
Crossref
Maria Pia CicaleseAlessandro Aiuti. (2015) Clinical Applications of Gene Therapy for Primary Immunodeficiencies. Human Gene Therapy 26:4, pages 210-219.
Crossref
Crossref
Aisha V. Sauer, Biagio Di Lorenzo, Nicola Carriglio & Alessandro Aiuti. (2014) Progress in gene therapy for primary immunodeficiencies using lentiviral vectors. Current Opinion in Allergy & Clinical Immunology 14:6, pages 527-534.
Crossref
Crossref
Marco Ranzani, Stefano Annunziato, David J. Adams & Eugenio Montini. (2013) Cancer Gene Discovery: Exploiting Insertional Mutagenesis. Molecular Cancer Research 11:10, pages 1141-1158.
Crossref
Crossref
Sayandip Mukherjee & Adrian J. Thrasher. (2013) Gene therapy for PIDs: Progress, pitfalls and prospects. Gene 525:2, pages 174-181.
Crossref
Crossref
Nizar MahlaouiIsabelle PellierCécile MignotJean-Philippe JaisChrystèle Bilhou-NabéraDespina MoshousBénédicte NevenCapucine PicardGeneviève de Saint-BasileMarina Cavazzana-Calvo, Stéphane BlancheAlain Fischer. (2013) Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome. Blood 121:9, pages 1510-1516.
Crossref
Crossref
Samantha Scaramuzza, Luca Biasco, Anna Ripamonti, Maria C Castiello, Mariana Loperfido, Elena Draghici, Raisa J Hernandez, Fabrizio Benedicenti, Marina Radrizzani, Monica Salomoni, Marco Ranzani, Cynthia C Bartholomae, Elisa Vicenzi, Andrea Finocchi, Robbert Bredius, Marita Bosticardo, Manfred Schmidt, Christof von Kalle, Eugenio Montini, Alessandra Biffi, Maria G Roncarolo, Luigi Naldini, Anna Villa & Alessandro Aiuti. (2013) Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome. Molecular Therapy 21:1, pages 175-184.
Crossref
Crossref
Michelle P. Lambert & Mortimer Poncz. 2013. Platelets. Platelets
971
987
.
Lajos Baranyi & Boro Dropulic. 2013. Mesenchymal Stem Cell Therapy. Mesenchymal Stem Cell Therapy
271
320
.
Alessandro Aiuti, Rosa Bacchetta, Reinhard Seger, Anna Villa & Marina Cavazzana-Calvo. (2012) Gene therapy for primary immunodeficiencies: Part 2. Current Opinion in Immunology 24:5, pages 585-591.
Crossref
Crossref
Anne Galy & Adrian J. Thrasher. (2011) Gene therapy for the Wiskott–Aldrich syndrome. Current Opinion in Allergy & Clinical Immunology 11:6, pages 545-550.
Crossref
Crossref
Inés Avedillo Díez, Daniela Zychlinski, Emanuele G. Coci, Melanie Galla, Ute Modlich, Ricardo A. Dewey, Adrian Schwarzer, Tobias Maetzig, Nonsikelelo Mpofu, Elmar Jaeckel, Kaan Boztug, Christopher Baum, Christoph Klein & Axel Schambach. (2011) Development of Novel Efficient SIN Vectors with Improved Safety Features for Wiskott–Aldrich Syndrome Stem Cell Based Gene Therapy. Molecular Pharmaceutics 8:5, pages 1525-1537.
Crossref
Crossref
Laurence Jeanson-Leh, Sabine Charrier, Alexis Proust, Chrystele Bilhou-Nabéra, Rémi Favier, Caroline Deswarte, Pierre Bordigoni, Anne Galy & Jean Delaunay. (2011) The c.273+11dup genetic change in the WAS gene is a functionally neutral polymorphism. European Journal of Haematology 87:4, pages 366-371.
Crossref
Crossref
Marita Bosticardo, Elena Draghici, Francesca Schena, Aisha Vanessa Sauer, Elena Fontana, Maria Carmina Castiello, Marco Catucci, Michela Locci, Luigi Naldini, Alessandro Aiuti, Maria Grazia Roncarolo, Pietro Luigi Poliani, Elisabetta Traggiai & Anna Villa. (2011) Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome. Journal of Allergy and Clinical Immunology 127:6, pages 1376-1384.e5.
Crossref
Crossref
S Charrier, M Ferrand, M Zerbato, G Précigout, A Viornery, S Bucher-Laurent, S Benkhelifa-Ziyyat, O W Merten, J Perea & A Galy. (2010) Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction. Gene Therapy 18:5, pages 479-487.
Crossref
Crossref
Otto-Wilhelm MertenSabine CharrierNicolas LaroudieSylvain FauchilleCéline DuguéChristine JennyMuriel AuditMaria-Antonietta Zanta-BoussifHélène ChautardMarina RadrizzaniGiuliana VallantiLuigi NaldiniPatricia Noguiez-HellinAnne Galy. (2011)
Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical
Ex Vivo
Gene Therapy Application
. Human Gene Therapy 22:3, pages 343-356.
Crossref
Crossref
Crystal J. MacKenzie & Toshi Shioda. (2011) COS‐1 Cells as Packaging Host for Production of Lentiviruses. Current Protocols in Cell Biology 50:1.
Crossref
Crossref
Manuel Grez, Janine Reichenbach, Joachim Schwäble, Reinhard Seger, Mary C Dinauer & Adrian J Thrasher. (2011) Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma. Molecular Therapy 19:1, pages 28-35.
Crossref
Crossref
Patrice P. Denèfle. 2011. Viral Vectors for Gene Therapy. Viral Vectors for Gene Therapy
27
44
.
Janka Mátrai, Marinee KL Chuah & Thierry VandenDriessche. (2010) Recent Advances in Lentiviral Vector Development and Applications. Molecular Therapy 18:3, pages 477-490.
Crossref
Crossref
Maheswaran ManiShivkumar Venkatasubrahmanyam, Mrinmoy Sanyal, Shoshana Levy, Atul Butte, Kenneth WeinbergThomas Jahn. (2009) Wiskott-Aldrich syndrome protein is an effector of Kit signaling. Blood 114:14, pages 2900-2908.
Crossref
Crossref
Alessandro Aiuti, Immacolata Brigida, Francesca Ferrua, Barbara Cappelli, Robert Chiesa, Sarah Marktel & Maria-Grazia Roncarolo. (2009) Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID. Immunologic Research 44:1-3, pages 150-159.
Crossref
Crossref
Pamela P. W. Lee, Tong-Xin Chen, Li-Ping Jiang, Jing Chen, Koon-wing Chan, Tze-Leung Lee, Marco H. K. Ho, Shao-Han Nong, Yin Yang, Yong-Jun Fang, Qiang Li, Xiao-Chun Wang, Xi-Qiang Yang & Yu-Lung Lau. (2009) Clinical and Molecular Characteristics of 35 Chinese Children with Wiskott–Aldrich Syndrome. Journal of Clinical Immunology 29:4, pages 490-500.
Crossref
Crossref
Marita BosticardoFrancesco MarangoniAlessandro Aiuti, Anna Villa & Maria Grazia Roncarolo. (2009) Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome. Blood 113:25, pages 6288-6295.
Crossref
Crossref
Shannon L. Smith & Toshi Shioda. (2009) Advantages of COS-1 monkey kidney epithelial cells as packaging host for small-volume production of high-quality recombinant lentiviruses. Journal of Virological Methods 157:1, pages 47-54.
Crossref
Crossref
Alessandro Aiuti & Maria Grazia Roncarolo. (2009) Ten years of gene therapy for primary immune deficiencies. Hematology 2009:1, pages 682-689.
Crossref
Crossref
Adrian J. Thrasher. (2009) New insights into the biology of Wiskott-Aldrich syndrome (WAS). Hematology 2009:1, pages 132-138.
Crossref
Crossref
Lisa S. WesterbergMiguel A. de la FuenteFredrik Wermeling, Hans D. Ochs, Mikael C. I. Karlsson, Scott B. SnapperLuigi D. Notarangelo. (2008) WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function. Blood 112:10, pages 4139-4147.
Crossref
Crossref
Adrian J. Thrasher. (2008) Gene Therapy for Primary Immunodeficiencies. Immunology and Allergy Clinics of North America 28:2, pages 457-471.
Crossref
Crossref