Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Science Translational Medicine ◽

10.1126/scitranslmed.aad8856 ◽

2016 ◽

Vol 8 (335) ◽

pp. 335ra57-335ra57 ◽

Author(s):

Suk See De Ravin ◽

Xiaolin Wu ◽

Susan Moir ◽

Lela Kardava ◽

Sandra Anaya-O’Brien ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Erratum for the Research Article: “Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency” by S. S. De Ravin, X. Wu, S. Moir, S. Anaya-O’Brien, N. Kwatemaa, P. Littel, N. Theobald, U. Choi, L. Su, M. Marquesen, D. Hilligoss, J. Lee, C. M. Buckner, K. A. Zarember, G. O’Connor, D. McVicar, D. Kuhns, R. E. Throm, S. Zhou, L. D. Notarangelo, I. C. Hanson, M. J. Cowan, E. Kang, C. Hadigan, M. Meagher, J. T. Gray, B. P. Sorrentino, H. L. Malech

Science Translational Medicine ◽

10.1126/scitranslmed.aag1383 ◽

2016 ◽

Vol 8 (341) ◽

pp. 341er5-341er5

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Hematopoietic Stem ◽

Research Article ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Reconstitution of immune function in adenosine deaminase deficient severe combined immunodeficiency following hematopoietic stem cell gene therapy

Blood Cells Molecules and Diseases ◽

10.1016/j.bcmd.2007.10.041 ◽

2008 ◽

Vol 40 (2) ◽

pp. 266-267

Author(s):

H. Bobby Gaspar ◽

Emma Bjorkegren ◽

Kate Parsley ◽

Kimberly Gilmour ◽

Lynette Fairbanks ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Immune Function ◽

Adenosine Deaminase ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Faculty Opinions recommendation of Repetitive busulfan administration after hematopoietic stem cell gene therapy associated with a dominant HDAC7 clone in a nonhuman primate.

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

10.3410/f.4242977.4039078 ◽

2010 ◽

Author(s):

Karen E Pollok ◽

Shanbao Cai

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Nonhuman Primate ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Faculty Opinions recommendation of Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.

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

10.3410/f.720388464.793515025 ◽

2016 ◽

Author(s):

Fabio Candotti

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Hematopoietic Stem ◽

Immunological Recovery ◽

Metabolic Correction ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.263 ◽

2021 ◽

Vol 132 (2) ◽

pp. S107

Author(s):

Niek P. van Til ◽

Yildirim Dogan ◽

Cecilia Barese ◽

Zeenath Unnisa ◽

Swaroopa Guda ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Preclinical Study ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Update on safety and efficacy of lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD)

European Journal of Paediatric Neurology ◽

10.1016/j.ejpn.2017.04.986 ◽

2017 ◽

Vol 21 ◽

pp. e20

Author(s):

F. Fumagalli ◽

V. Calbi ◽

A. Zambon ◽

F. Ciotti ◽

L. Lorioli ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Metachromatic Leukodystrophy ◽

Hematopoietic Stem ◽

Safety And Efficacy ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy

Hematology/Oncology Clinics of North America ◽

10.1016/j.hoc.2017.06.006 ◽

2017 ◽

Vol 31 (5) ◽

pp. 721-735 ◽

Author(s):

Donald B. Kohn

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Historical Perspective ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Progress in Hematopoietic Stem Cell Gene Therapy II

Molecular Therapy ◽

10.1006/mthe.2001.0382 ◽

2001 ◽

Vol 3 (5) ◽

pp. S155-S157

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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494. Hematopoietic Stem Cell Gene Therapy for Cystinosis

Molecular Therapy ◽

10.1016/s1525-0016(16)34829-8 ◽

2013 ◽

Vol 21 ◽

pp. S190-S191

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

Download Full-text

Development of a modified selective amplifier gene for hematopoietic stem cell gene therapy

Gene Therapy ◽

10.1038/sj.gt.3300906 ◽

1999 ◽

Vol 6 (6) ◽

pp. 1038-1044 ◽

Author(s):

K M Matsuda ◽

A Kume ◽

Y Ueda ◽

M Urabe ◽

M Hasegawa ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Selective Amplifier ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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