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Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.

• Authors : Braun CJ; Dr. von Hauner Children's Hospital, Ludwig Maximilians University Munich, 80337 Munich, Germany.; Boztug K

Subjects: Genetic Therapy/Genetic Therapy/Genetic Therapy/*adverse effects ; Mutagens/Mutagens/Mutagens/*adverse effects ; Wiskott-Aldrich Syndrome/Wiskott-Aldrich Syndrome/Wiskott-Aldrich Syndrome/*therapy

• Source: Science translational medicine [Sci Transl Med] 2014 Mar 12; Vol. 6 (227), pp. 227ra33.Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication

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Nuclear role of WASp in the pathogenesis of dysregulated TH1 immunity in human Wiskott-Aldrich syndrome.

• Authors : Taylor MD; Division of Pediatric Hematology-Oncology, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, PA 15213, USA.; Sadhukhan S

Subjects: Cell Nucleus/Cell Nucleus/Cell Nucleus/*metabolism ; Immunity/Immunity/Immunity/*immunology ; Th1 Cells/Th1 Cells/Th1 Cells/*immunology

• Source: Science translational medicine [Sci Transl Med] 2010 Jun 23; Vol. 2 (37), pp. 37ra44.Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication

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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

• Authors : Aiuti A; San Raffaele Telethon Institute for Gene Therapy, Division of Regenerative Medicine, Stem Cells, and Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy. ; Biasco L

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

• Source: Science (New York, N.Y.) [Science] 2013 Aug 23; Vol. 341 (6148), pp. 1233151. Date of Electronic Publication: 2013 Jul 11.Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 0404511 Publication

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The many faces of WAS protein.

• Authors : Featherstone C

Subjects: Signal Transduction*; Actins/Actins/Actins/*metabolism ; Cell Cycle Proteins/Cell Cycle Proteins/Cell Cycle Proteins/*metabolism

• Source: Science (New York, N.Y.) [Science] 1997 Jan 03; Vol. 275 (5296), pp. 27-8.Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 0404511 Publication

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A human DCC variant causing mirror movement disorder reveals that the WAVE regulatory complex mediates axon guidance by netrin-1–DCC.

• Authors : Chaudhari, Karina; Zhang, Kaiyue; Yam, Patricia T.

Subjects: WISKOTT-Aldrich syndrome; AXONS; CONGENITAL disorders

• Source: Science Signaling; 10/1/2024, Vol. 17 Issue 856, p1-17, 17p

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Medicine. Gene therapy that works.

• Authors : Verma IM; Laboratory of Genetics, The Salk Institute, La Jolla, CA 92037, USA.

Subjects: Hematopoietic Stem Cell Transplantation*; Cerebroside-Sulfatase/Cerebroside-Sulfatase/Cerebroside-Sulfatase/*genetics ; Genetic Therapy/Genetic Therapy/Genetic Therapy/*methods

• Source: Science (New York, N.Y.) [Science] 2013 Aug 23; Vol. 341 (6148), pp. 853-5.Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 0404511 Publication

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Education Full Text (H.W. Wilson)

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Education Full Text (H.W. Wilson)

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Hereditary spastic paraplegia SPG8 mutations impair CAV1-dependent, integrin-mediated cell adhesion.

• Authors : Lee, Seongju; Park, Hyungsun; Zhu, Peng-Peng

Subjects: INTEGRINS; FAMILIAL spastic paraplegia; CELL adhesion

• Source: Science Signaling; 1/7/2020, Vol. 13 Issue 613, p1-12, 12p

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Education Full Text (H.W. Wilson)

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