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Cited by 59

The DOI matches its Crossref record; 45 of the 45 references it cites were found in a registry or an index.

Development of Novel Efficient SIN Vectors with Improved Safety Features for Wiskott–Aldrich Syndrome Stem Cell Based Gene Therapy

Avedillo Díez, Inés, Zychlinski, Daniela, Coci, Emanuele G., Galla, Melanie, Modlich, Ute, Dewey, Ricardo A., Schwarzer, Adrian, Maetzig, Tobias, Mpofu, Nonsikelelo, Jaeckel, Elmar, Boztug, Kaan, Baum, Christopher, Klein, Christoph, Schambach, Axel·2011·Molecular Pharmaceutics
DOI 10.1021/mp200132u
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Abstract

Gene therapy is a promising therapeutic approach to treat primary immunodeficiencies. Indeed, the clinical trial for the Wiskott-Aldrich Syndrome (WAS) that is currently ongoing at the Hannover Medical School (Germany) has recently reported the correction of all affected cell lineages of the hematopoietic system in the first treated patients. However, an extensive study of the clonal inventory of those patients reveals that LMO2, CCND2 and MDS1/EVI1 were preferentially prevalent. Moreover, a first leukemia case was observed in this study, thus reinforcing the need of developing safer vectors for gene transfer into HSC in general. Here we present a novel self-inactivating (SIN) vector for the gene therapy of WAS that combines improved safety features. We used the elongation factor 1 alpha (EFS) promoter, which has been extensively evaluated in terms of safety profile, to drive a codon-optimized human WASP cDNA. To test vector performance in a more clinically relevant setting, we transduced murine HSPC as well as human CD34+ cells and also analyzed vector efficacy in their differentiated myeloid progeny. Our results show that our novel vector generates comparable WAS protein levels and is as effective as the clinically used LTR-driven vector. Therefore, the described SIN vectors appear to be good candidates for potential use in a safer new gene therapy protocol for WAS, with decreased risk of insertional mutagenesis.

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Research works printed but not linked: 4

  1. Boztug K., 2006, Curr. Opin. Mol. Ther., 8, 390
  2. Itoh K., 1989, Exp. Hematol., 17, 145
  3. IDW Press Release:Effective gene therapy for children with Wiskott-Aldrich-syndrome. Nov 11, 2010.
  4. Modlich U., 2009, Mol. Ther., 17, 1919, 10.1038/mt.2009.179 · DOI · Paper page

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Metadata

Authors
Avedillo Díez, I., Zychlinski, D., Coci, E. G., Galla, M., Modlich, U., Dewey, R. A., Schwarzer, A., Maetzig, T., Mpofu, N., Jaeckel, E., Boztug, K., Baum, C., Klein, C., & Schambach, A.
Affiliations
Department of Pediatric Hematology/Oncology, ∥Institute of Experimental Hematology, ‡Excellence Cluster REBIRTH, ⊥Integrated Research and Treatment Center Transplantation, and #Department of Gastroenterology, Hepatology and Endocrinology, Hannover Medical School, Germany
Venue
Molecular Pharmaceutics
Publisher
American Chemical Society (ACS)
Published
2011-08-31
Volume
8
Issue
5
Pages
1525-1537
ISSN
  • 1543-8384
  • 1543-8392
DOI
10.1021/mp200132u
Cited by
59
References
45
Language
en

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The paper
Avedillo Díez, I., Zychlinski, D., Coci, E. G., Galla, M., Modlich, U., Dewey, R. A., Schwarzer, A., Maetzig, T., Mpofu, N., Jaeckel, E., Boztug, K., Baum, C., Klein, C., & Schambach, A. (2011). Development of Novel Efficient SIN Vectors with Improved Safety Features for Wiskott–Aldrich Syndrome Stem Cell Based Gene Therapy. Molecular Pharmaceutics. https://doi.org/10.1021/mp200132u

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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).

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