Evaluation of engraftment and immunological tolerance after reduced intensity conditioning in a rhesus hematopoietic stem cell gene therapy model-Reference-Cited by-同舟云学术

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Evaluation of engraftment and immunological tolerance after reduced intensity conditioning in a rhesus hematopoietic stem cell gene therapy model

Published:2013-11-21 Issue:2 Volume:21 Page:148-157
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Uchida N,Weitzel R P,Evans M E,Green R,Bonifacino A C,Krouse A E,Metzger M E,Hsieh M M,Donahue R E,Tisdale J F

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/gt201367.pdf

Reference35 articles.

1. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360: 447–458.

2. Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410–2413.

3. Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 2010; 467: 318–322.

4. Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F, Wulffraat N, McIntyre E et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348: 255–256.

5. Schwarzwaelder K, Howe SJ, Schmidt M, Brugman MH, Deichmann A, Glimm H et al. Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest 2007; 117: 2241–2249.

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1. Nestin-GFP transgene labels immunoprivileged bone marrow mesenchymal stem cells in the model of ectopic foci formation;Frontiers in Cell and Developmental Biology;2022-09-05

2. Immunological barriers to haematopoietic stem cell gene therapy;Nature Reviews Immunology;2022-03-17

3. Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models;Cell Reports Medicine;2021-04

4. Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies;Cell Stem Cell;2021-02

5. Immunoresponse to Gene-Modified Hematopoietic Stem Cells;Molecular Therapy - Methods & Clinical Development;2020-03

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