Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice

C. Klein

doi:10.1182/blood-2002-05-1423

Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice

Blood ◽

10.1182/blood-2002-05-1423 ◽

2002 ◽

Vol 101 (6) ◽

pp. 2159-2166 ◽

Cited By ~ 88

Author(s):

C. Klein

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

T Cell ◽

Cell Signaling ◽

Hematopoietic Stem Cells ◽

T Cell Signaling ◽

Hematopoietic Stem ◽

Wiskott Aldrich Syndrome

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Potential use of T cell receptor genes to modify hematopoietic stem cells for the gene therapy of cancer

Pathology & Oncology Research ◽

10.1053/paor.1999.0003 ◽

1999 ◽

Vol 5 (1) ◽

pp. 3-15 ◽

Cited By ~ 16

Author(s):

Timothy M Clay ◽

Mary C Custer ◽

Paul J Spiess ◽

Michael I Nishimura

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

T Cell ◽

Hematopoietic Stem Cells ◽

T Cell Receptor ◽

Cell Receptor ◽

Hematopoietic Stem ◽

T Cell Receptor Genes ◽

Potential Use

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345. Gene Therapy for Wiskott-Aldrich Syndrome Using Lentiviral Vectors: Evidence for Efficacy and Safety after Transduction of Human T Cells and Hematopoietic Stem Cells

Molecular Therapy ◽

10.1016/j.ymthe.2005.06.348 ◽

2005 ◽

Vol 11 ◽

pp. S134-S135

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

T Cells ◽

Hematopoietic Stem Cells ◽

Lentiviral Vectors ◽

Efficacy And Safety ◽

Hematopoietic Stem ◽

Wiskott Aldrich Syndrome ◽

Human T Cells

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987. Lentiviral Transduction of the Human Purine Nucleoside Phosphorylase (PNP) Gene in Murine Hematopoietic Stem Cells: An In Vivo Model of Gene Therapy for PNP- T-Cell Immunodeficiency

Molecular Therapy ◽

10.1016/s1525-0016(16)39345-5 ◽

2009 ◽

Vol 17 ◽

pp. S377

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

T Cell ◽

Hematopoietic Stem Cells ◽

Purine Nucleoside Phosphorylase ◽

In Vivo Model ◽

Hematopoietic Stem ◽

Lentiviral Transduction ◽

T Cell Immunodeficiency

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390. Correction of the Deficient Secondary T-Cell Response to Influenza Virus in Wiskott Aldrich Syndrome (WAS) Knockout Mice by Transplantation of Syngeneic Hematopoietic Stem Cells Transduced with a WASP-Expressing Retroviral Vector

Molecular Therapy ◽

10.1016/s1525-0016(16)40832-4 ◽

2003 ◽

Vol 7 (5) ◽

pp. S153

Keyword(s):

Stem Cells ◽

Influenza Virus ◽

T Cell ◽

Hematopoietic Stem Cells ◽

Knockout Mice ◽

Cell Response ◽

Retroviral Vector ◽

T Cell Response ◽

Hematopoietic Stem ◽

Wiskott Aldrich Syndrome

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Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA

Case Medical Research ◽

10.31525/ct1-nct04201405 ◽

2019 ◽

Author(s):

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Mucopolysaccharidosis Type ◽

Hematopoietic Stem ◽

Type Iiia ◽

Mucopolysaccharidosis Type Iiia

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Faculty Opinions recommendation of Fetal and adult hematopoietic stem cells give rise to distinct T cell lineages in humans.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.7455956.8916054 ◽

2011 ◽

Author(s):

Juan Carlos Zúñiga-Pflücker ◽

Patricia Benveniste

Keyword(s):

Stem Cells ◽

T Cell ◽

Hematopoietic Stem Cells ◽

Hematopoietic Stem ◽

Cell Lineages

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Genetic Deletion of CD7 in Hematopoietic Stem Cells to Restore Immunity after CAR T Cell Therapy for T Cell Malignancies

Transplantation and Cellular Therapy ◽

10.1016/s2666-6367(21)00100-7 ◽

2021 ◽

Vol 27 (3) ◽

pp. S71-S72

Author(s):

Miriam Y. Kim ◽

Matthew Cooper ◽

Julie K. Ritchey ◽

Julia Hollaway ◽

John F. DiPersio

Keyword(s):

Stem Cells ◽

T Cell ◽

Cell Therapy ◽

Hematopoietic Stem Cells ◽

T Cell Therapy ◽

Hematopoietic Stem ◽

Car T Cell Therapy ◽

Car T ◽

Genetic Deletion ◽

T Cell Malignancies

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3040 – IN VITRO RECAPITULATION OF MURINE T CELL DEVELOPMENT FROM SINGLE HEMATOPOIETIC STEM CELLS

Experimental Hematology ◽

10.1016/j.exphem.2020.09.060 ◽

2020 ◽

Vol 88 ◽

pp. S51

Author(s):

Victoria Sun ◽

Amelie Montel-Hagen ◽

David Casero ◽

Steven Tsai ◽

Alexandre Zampieri ◽

...

Keyword(s):

Stem Cells ◽

T Cell ◽

Hematopoietic Stem Cells ◽

T Cell Development ◽

Cell Development ◽

Hematopoietic Stem

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Development of gene therapy for hematopoietic stem cells

Scandinavian Journal of Clinical and Laboratory Investigation ◽

10.1080/003655102320754700 ◽

2002 ◽

Vol 62 (5) ◽

pp. 10-10

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

Hematopoietic Stem Cells ◽

Hematopoietic Stem

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Faculty Opinions recommendation of Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.739368425.793582178 ◽

2021 ◽

Author(s):

Frank Staal

Keyword(s):

Stem Cells ◽

Gene Therapy ◽

Mouse Model ◽

Hematopoietic Stem Cells ◽

Cord Blood ◽

Lentiviral Vector ◽

Hematopoietic Stem ◽

Diamond Blackfan Anemia ◽

Human Cd34

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