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Bone Marrow & Cord Blood Transplant
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Literature
Medical Resources
- Medical Literature
- Review Articles
- New insights into the biology of WAS, A Thrasher, Hematology 2009
- Wiskott-Aldrich Syndrome by Dr. S. Burns and Dr. A. Thrasher. British Medical Journal
- Wisk0tt-Aldrich Syndrome. National Library of Medicine
- Wiskott Aldrich Syndrome from Socialstyrelsen by Anders Fasth, M.D.
- Wiskott Aldrich Syndrome Family Handbook from the Immune Deficiency Foundation
- Wiskott Aldrich Syndrome from Patient Plus UK
- Wiskott-Aldrich Syndrome, Donald Dibbern, e-medicine
- Wiskott-Aldrich Syndrome: Diagnosis, Clinical and Laboratory Manifestations, and Treatment Hans D. Ochs Biology of Blood and Marrow transplantation Volume 15, Issue 1, Supplement, Pages 84-90 (January 2009)
- WAS Related Disorders Filipovich, A et al Gene Reviews Sept. 2004
- Wiskott Aldrich Syndrome-A multi institutional survey
- Databases
- Disease Database listing 294 mutations from Resource of Asian Primary Immunodeficiencies(RAPID)
- Diseases Database: Wiskott-Aldrich Syndrome
- Wiskott-Aldrich Syndrome: WAS OMIM
- WASPBase Database of Published WAS gene mutations 2004
- Classification and Scoring of WAS
- Wiskott-Aldrich Syndrome: Diagnosis, Clinical and Laboratory Manifestations, and Treatment Hans D. Ochs, Alexandra H. Filipovich, Paul Veys, Morton J. Cowan, Neena Kapoor Biology of Blood and Marrow Transplantation Volume 15, Issue 1, Supplement, Pages 84-90 (January 2009)
- Clinical Phenotypes of Wiskott Aldrich Syndrome: Notarangelo LD et al Current Opinion in Hematology Jan 2008
- Clinical Scoring of patients with WAS Jin Y et al Blood Dec 15 2004
- Scoring based on Wiskott Aldrich Syndrome Protein(WASP) Levels: Shcherbina A, Journal Of Immunology, Dec. 1 1999
- Clinical Course of Patients
- X-Linked Thrombocytopenia due to WAS Mutations: Clinical Characteristics, long-term outcome, and treatment options, Michael Albert et al, Blood First Line Edition Paper, prepublished February 19, 2010
- Clinical Course of Patients with WASP Gene Mutations, Imai, K et al Blood, 15 Jan. 2004, Vol.103, No.2, pp.456-464
- Autoimmunity in WAS
- Autoimmunity in Wiskott-Aldrich Syndrome: Risk Factors, Clinical features, and Outcome in a Single-Center Cohort of 55 Patients Sophie Dupuis-Girod, MD et al Pediatrics Vol 111 No. 5 May 2003, ppe622-e627
- Abstract: Autoimmunity in Wiskott-Aldrich Syndrome Schurman, SH et al Current Opinion in Rheumatology, 2003 Jul, 15(4):446-53
- Abstract: Autoimmune disroders and lymphoma: A multiinstitutional survey of the Wiskott-Aldrich syndrome. Sullivan, KE J Pediatrics1994 Dec;125(6 Pt 1): 876-8
- Genotype-Phenotype Correlations
- Mutations of the Wiskott-Aldrich Syndrome Protein(WASP): hotspots, effect on transcription, and translation and phenotype/genotype correlation Jin Y et al Blood, 15 Dec 2004, Vol. No. 13, pp. 4010-4019
- Abstract: Wiskott-Aldrich Syndrome: a model for defective actin reorganization, cell trafficking and synapse formation. Notarangelo, LD Current Opinion in Immunology: 2003 Oct;15(5):585-91
- Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia: WASP Gene Mutations, Protein Expression and Phenotype Zhu, Q et al Blood, Vo. 90 No. 7(Oct. 1), 1997: pp. 2680-2689
- Treatment Options
- An international study examining therapeutic options used in treatment of Wiskott-Aldrich syndrome. Conley ME et al JLClin Immunol. 2003; 109: 272–7. [PubMed]
- Transplants
- Wiskott Aldrich Syndrome and Transplant - NMDP
- Abstract: Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich Syndrome:collaborative study of the European Society for Immunodeficiencies and European Grop for Blood and Marrow Transplantation Ozsahin, H et al Blood, 1 January 2008, Vol.111, No. 1, pp.439-445
- Results and long term outcome in 39 patients with Wiskott-Aldrich Syndrome transplanted from HLA matched and mismatched donors Wilhelm Friedrich et al Immmunologic Research Oct.2008
- Abstract: Hematopoietic cell transplantation for correction of primary immunodeficienciesFilipovich A. Bone Marrow Transplant 2008 Aug;42 Suppl 1:S49-S52.
- Abstract: Stem Cell Transplantation for the Wiskott-Aldrich Syndrome: a single-center experience confirms efficacy of matched unrelated donor transplantation Pai SY et al Pediatr Hematol Oncol. 2007 Sep;24(6):393-402.
- Unmatched Bone Marrow Transplant Can Be As Effective As A Perfect Match in Young Children Filipovich A Cincinnati Children's Hospital
- Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich Syndrome: collaborative study of teh International Bone Marrow Transplant Registry and the National Marrow Donor Program, Blood, mar 15 2001
- Prevention of Opportunistic Infections in HSCT Recipients MMWR, Oct 20, 2000
- Umbilical Cord Blood Transplant in Wiskott Aldrich Syndrome Knutsen A. et al Volume 142,Issue 5, Pages 519-523 (May 2003)
- Preventing Opportunistic infections in HSCT Recipients-An excellent review article from the CDC which includes how to prevent infections, immunization etc.
- Gene Therapy
- Gene Therapy for Immunodeficiency due to Adenosine Deaminase Deficiency Alessandro Aiuti, Federica Cattaneo, Stefania Galimberti, Ulrike Benninghoff, Barbara Cassani, Luciano Callegaro, Samantha Scaramuzza,, Grazia Andolfi, Massimiliano Mirolo, Immacolata Brigida, Antonella Tabucchi, Filippo Carlucci, Martha Eibl, Memet Aker,Shimon Slavin, Hamoud Al-Mousa, Abdulaziz Al Ghonaium, Alina Ferster, Andrea Duppenthaler, Luigi Notarangelo, Uwe Wintergerst, Rebecca H. Buckley, Marco Bregni, Sarah Marktel,Maria Grazia Valsecchi, Paolo Rossi, Fabio Ciceri, Roberto Miniero, Claudio Bordignon and Maria-Grazia Roncarolo, M.D. Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. The New England Journal of Medicine Volume, Jan 29, 2009 ; 360:447-458
- Development of lentiviral gene therapy for Wiskott Aldrich syndrome Anne Galy, Maria-Grazia Roncarolo & Adrian J Thrasher
- Expert Opinion on Biological Therapy. February 2008, Vol. 8, No. 2, Pages 181-190
- Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome, Session Type: Oral Boztug, K et al Proceedings of American Society of Hematology Meeting, Monday, December 10, 2007
- Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in miceChristoph Klein Blood, 15 March 2003, Vol. 101, No. 6, pp. 2159-2166
- Revertant Changes in WAS Patients
- Multiple independent second-site mutations in two siblings with somatic mosaicism for Wiskott-Aldrich Syndrome Clin Genet. 2008 Jul;74(1):68-74 Boztug K, Germeshausen M, Avedillo Diez I, Gulacsy V, Diestelhorst J, Ballmaier M, Welte K, marodi L, Chernyshova L, Klein C
- Multiple patients with revertant mosaicism in a single Wiskott-Aldrich Syndrome Family Wada, T et al Blood, 1 Sept. 2004, Vol. 104, No. 5, pp.1270-1272
- Evolution of Highly Polymorhic T Cell Populations in Siblings with the Wiskott-Aldrich Syndrome Lutskiy, M et al
- Somatic Mosaicism in Wiskott-Aldrich Syndrome suggests in vivo reversion by a DNA slippage mechanism Wada, T et al Proceedings of the National Academy of Sciences of the U.S.A.
- The Phenomenon of SpontaneousGenetic Reversions in the Wiskott-Aldrich Syndrome Donn M. Stewart et al Journal of Clinical Immunology, Nov. 2007