The DOI matches its Crossref record; 32 of the 33 references it cites were found in a registry or an index.
Sustainable correction of junctional epidermolysis bullosa via transposon‐mediated nonviral gene transfer
Abstract
Sustainable correction of severe human genetic disorders of self-renewing tissues, such as the blistering skin disease junctional epidermolysis bullosa (JEB), is facilitated by stable genomic integration of therapeutic genes into somatic tissue stem cells. While integrating viral vectors can achieve this, they suffer from logistical and biosafety concerns. To circumvent these limitations, we used the Sleeping Beauty transposable element to integrate the LAMB3 cDNA into genomes of epidermal holoclones from six unrelated JEB patients. These cells regenerate human JEB skin that is normalized at the level of laminin 5 protein expression, hemidesmosome formation and blistering. Transposon-mediated gene delivery therefore affords an opportunity for stable gene delivery in JEB and other human diseases.
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Benedetti, S., Cossu, G., & Tedesco, F. S. (2015) · CRC Press eBooks · doi:10.1201/b18002-46 · Cited by 1
DNA Elements Tetris: A Strategy for Gene Correction
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Nonviral Gene Therapy—The Challenge of Mobilizing DNA
Mikkelsen, J. G. (2015) · Springer eBooks · doi:10.1007/978-1-4939-2389-2_4
Sleeping Beauty Transposon‐Mediated Stable Gene Delivery
Wilber, A. C., Zhou, X., Huang, X., Kaufman, D. S., & McIvor, R. S. (2011) · Wiley eBooks · doi:10.1002/9781118147177.ch8
Titeux, M., & Hovnanian, A. (2011) · Wiley eBooks · doi:10.1002/9781444345384.ch140
Regulated complex assembly protects cells from aberrant Sleeping Beauty transposition events
Pryputniewicz-Drobińska, D. (2010) · edoc Publication server (Humboldt University of Berlin) · doi:10.18452/16217
Current approaches to cutaneous gene therapy
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Gene therapeutic strategies for blistering skin diseases
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Characterization of the Sleeping Beauty transposon system for gene therapy applications
Score, P. R. (2008) · University of Minnesota Digital Conservancy (University of Minnesota)
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Review Skin gene therapy for acquired and inherited disorders
Carretero, M., Prada, F., Mirones, I., García, M., Holguín, A., Duarte, B., Podhajcer, O. L., Larcher, F., & Río, M. D. (2006)
Raisingtheroofonepidermolysisbullosa(EB): afocusonnewtherapies
Kopecki, Z., & Cowin, A. J. (2009)
Woodard, L. E., & Calos, M. P. (2015) · CRC Press eBooks · doi:10.1201/b18002-30
Davis, B. R., & Prokopishyn, N. L. (2009) · Elsevier eBooks · doi:10.1016/b978-0-12-374729-7.00065-2
Davis, B. R., & Prokopishyn, N. L. (2012) · Elsevier eBooks · doi:10.1016/b978-0-12-385942-6.00077-9
Basic Principles of Genetics and Gene Therapy
Kraemer, L., & Christiano, A. M. (2010) · Springer eBooks · doi:10.1007/978-3-540-78814-0_5
Metadata
- Authors
- Ortiz-Urda, S., Lin, Q., Yant, S. R., Keene, D., Kay, M. A., & Khavari, P. A.
- Affiliations
- VA Palo Alto Health Care System (US)
- Shriners Hospitals for Children - Portland (US)
- Stanford Medicine (US)
- Venue
- Gene Therapy
- Published
- 2003-06-16
- Volume
- 10
- Issue
- 13
- Pages
- 1099-1104
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- OpenAlex
- W2117888324
- Cited by
- 118
- References
- 33
- Citations by year
- 2020: 3 · 2019: 2 · 2017: 8 · 2016: 3 · 2015: 9 · 2014: 5 · 2013: 4 · 2012: 7
- FWCI
- 4.62
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cell Biology › Skin and Cellular Biology Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Animal Genetics and Reproduction
- Keywords
- junctional epidermolysis bullosa, Sleeping Beauty transposon, nonviral gene transfer, LAMB3, laminin-5, epidermal stem cells, skin regeneration, blistering skin disease
- MeSH terms
- Kalinin, Animals, Desmosomes, DNA Transposable Elements, Humans, Regeneration, Skin, Stem Cells, Genetic Therapy, Cell Adhesion Molecules, Epidermolysis Bullosa, Junctional, Microscopy, Immunoelectron, Mice, SCID, Mice
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