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Academic Journal

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome.

  • Authors : Rai R; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.; Romito M

Subjects: Gene Editing* ; Genetic Therapy*; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*metabolism

  • Source: Nature communications [Nat Commun] 2020 Aug 12; Vol. 11 (1), pp. 4034. Date of Electronic Publication: 2020 Aug 12.Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN:

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Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions.

  • Authors : Ferrua F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Marangoni F

Subjects: Genetic Therapy/Genetic Therapy/Genetic Therapy/*methods ; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*physiology ; Wiskott-Aldrich Syndrome/Wiskott-Aldrich Syndrome/Wiskott-Aldrich Syndrome/*therapy

  • Source: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2020 Aug; Vol. 146 (2), pp. 262-265. Date of Electronic Publication: 2020 Jul 02.Publisher: Mosby Country of Publication: United States NLM ID: 1275002 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-6825

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Academic Journal

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.

  • Authors : Ferrua F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods; Genetic Vectors/Genetic Vectors/Genetic Vectors/*genetics ; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*metabolism

  • Source: The Lancet. Haematology [Lancet Haematol] 2019 May; Vol. 6 (5), pp. e239-e253. Date of Electronic Publication: 2019 Apr 10.Publisher: Elsevier Ltd Country of Publication: England NLM ID: 101643584 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Academic Journal

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs.

  • Authors : Six E; INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, Paris, France.; Imagine Institute, Paris Descartes-Sorbonne Paris Cité University, Paris, France.

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Hematopoietic Stem Cell Transplantation*/Hematopoietic Stem Cell Transplantation*/Hematopoietic Stem Cell Transplantation*/methods; Clone Cells/Clone Cells/Clone Cells/*cytology

  • Source: Blood [Blood] 2020 Apr 09; Vol. 135 (15), pp. 1219-1231.Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print Cited Medium: Internet ISSN: 1528-0020

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Academic Journal

Effective management of acrodermatitis continua of Hallopeau with guselkumab in a Wiskott‐Aldrich syndrome patient.

Subjects: *HEMATOPOIETIC stem cell transplantation; *WISKOTT-Aldrich syndrome; *CORD blood

  • Source: International Journal of Dermatology. Sep2024, p1. 2p. 1 Illustration.

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Academic Journal

Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome.

  • Authors : Astrakhan A; Department of Immunology, University of Washington School of Medicine, Seattle, WA 98101, USA.; Sather BD

Subjects: Cell Lineage/Cell Lineage/Cell Lineage/*genetics ; Genetic Therapy/Genetic Therapy/Genetic Therapy/*methods ; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*metabolism

  • Source: Blood [Blood] 2012 May 10; Vol. 119 (19), pp. 4395-407. Date of Electronic Publication: 2012 Mar 19.Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Academic Journal

Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy.

Subjects: Genetic Vectors/Genetic Vectors/Genetic Vectors/*adverse effects ; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*metabolism ; Wiskott-Aldrich Syndrome/Wiskott-Aldrich Syndrome/Wiskott-Aldrich Syndrome/*metabolism

  • Source: Molecular pharmaceutics [Mol Pharm] 2011 Oct 03; Vol. 8 (5), pp. 1525-37. Date of Electronic Publication: 2011 Aug 31.Publisher: American Chemical Society Country of Publication: United States NLM ID: 101197791 Publication Model: Print-Electronic Cited Medium: Internet

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Academic Journal

Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.

  • Authors : Boztug K; Department of Pediatric Hematology/Oncology, Hannover Medical School, Carl-Neuberg-Strasse 1, D-30625 Hannover, Germany.; Dewey RA

Subjects: Genetic Therapy/Genetic Therapy/Genetic Therapy/*methods ; Hematopoietic Stem Cell Transplantation/Hematopoietic Stem Cell Transplantation/Hematopoietic Stem Cell Transplantation/*methods ; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*metabolism

  • Source: Current opinion in molecular therapeutics [Curr Opin Mol Ther] 2006 Oct; Vol. 8 (5), pp. 390-5.Publisher: Thomson Reuters (Scientific) Ltd Country of Publication: England NLM ID: 100891485 Publication Model: Print Cited Medium: Print ISSN:

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Academic Journal

A defect in hematopoietic stem cell migration explains the nonrandom X-chromosome inactivation in carriers of Wiskott-Aldrich syndrome.

  • Authors : Lacout C; U362 Institut National de la Santé et de la Recherche Médicale (INSERM), Institut Gustave Roussy, Villejuif, France.; Haddad E

Subjects: Dosage Compensation, Genetic*; Chemotaxis/Chemotaxis/Chemotaxis/*physiology ; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*cytology

  • Source: Blood [Blood] 2003 Aug 15; Vol. 102 (4), pp. 1282-9. Date of Electronic Publication: 2003 May 01.Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print-Electronic Cited Medium: Print ISSN: 0006-4971

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Academic Journal

Wiskott-Aldrich syndrome: a disorder of haematopoietic cytoskeletal regulation.

  • Authors : Thrasher AJ; Molecular Immunology Unit, Institute of Child Health, London WC1N 1EH, United Kingdom. ; Burns S

Subjects: Cytoskeletal Proteins*; Cytoskeleton/Cytoskeleton/Cytoskeleton/*pathology ; Hematopoietic Stem Cells/Hematopoietic Stem Cells/Hematopoietic Stem Cells/*pathology

  • Source: Microscopy research and technique [Microsc Res Tech] 1999 Oct 15; Vol. 47 (2), pp. 107-13.Publisher: Wiley-Liss Country of Publication: United States NLM ID: 9203012 Publication Model: Print Cited Medium: Print ISSN: 1059-910X (Print)

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  • 1-10 of  494 results for ""WISKOTT-Aldrich syndrome""