Gene therapy in combination with tissue engineering to treat epidermolysis bullosa - PubMed (original) (raw)
Review
Gene therapy in combination with tissue engineering to treat epidermolysis bullosa
Stefano Ferrari et al. Expert Opin Biol Ther. 2006 Apr.
Abstract
In the last 20 years epidermal stem cells have been extensively used for tissue regeneration of epidermis and other epithelial surfaces. The tremendous progress achieved has led to the development of protocols aimed at the correction of rare genetic disorders such as epidermolysis bullosa (EB), a severe, often lethal, blistering disorder of the skin. Approximately 400,000-500,000 people are affected worldwide and no definitive treatments have yet been developed. Gene therapy might represent an alternative therapeutic approach. This paper reviews the different strategies used to genetically modify keratinocytes from EB patients and addresses issues such as the use of in vivo or ex vivo approaches, how to target keratinocytes with stem cell properties in order to have long-term therapeutic gene expression, and which gene transfer agents should be used. The progress made has led the authors' group to submit a request for a Phase I/II ex vivo therapy clinical trial for patients with junctional EB.
Similar articles
- Towards a gene therapy clinical trial for epidermolysis bullosa.
Ferrari S, Pellegrini G, Matsui T, Mavilio F, De Luca M. Ferrari S, et al. Rev Recent Clin Trials. 2006 May;1(2):155-62. doi: 10.2174/157488706776876472. Rev Recent Clin Trials. 2006. PMID: 18473966 Review. - Gene therapy approaches for epidermolysis bullosa.
Ferrari S, Pellegrini G, Mavilio F, De Luca M. Ferrari S, et al. Clin Dermatol. 2005 Jul-Aug;23(4):430-6. doi: 10.1016/j.clindermatol.2004.07.017. Clin Dermatol. 2005. PMID: 16023940 Review. - Advances in Gene/Cell Therapy in Epidermolysis Bullosa.
Murauer EM, Koller U, Pellegrini G, De Luca M, Bauer JW. Murauer EM, et al. Keio J Med. 2015;64(2):21-5. doi: 10.2302/kjm.2014-0013-RE. Epub 2015 Jun 6. Keio J Med. 2015. PMID: 26050701 Review. - Treatment of hereditary epidermolysis bullosa: updates and future prospects.
Hsu CK, Wang SP, Lee JY, McGrath JA. Hsu CK, et al. Am J Clin Dermatol. 2014 Feb;15(1):1-6. doi: 10.1007/s40257-013-0059-z. Am J Clin Dermatol. 2014. PMID: 24402870 Review. - Stem Cell Therapy for Epidermolysis Bullosa-Does It Work?
Tamai K, Uitto J. Tamai K, et al. J Invest Dermatol. 2016 Nov;136(11):2119-2121. doi: 10.1016/j.jid.2016.07.004. J Invest Dermatol. 2016. PMID: 27772543 Review.
Cited by
- Gene Therapy and its Application in Dermatology.
Sarkar T, Sarkar S, Gangopadhyay DN. Sarkar T, et al. Indian J Dermatol. 2020 Sep-Oct;65(5):341-350. doi: 10.4103/ijd.IJD_323_20. Indian J Dermatol. 2020. PMID: 33165431 Free PMC article. Review. - Gene Delivery to Human Limbal Stem Cells Using Viral Vectors.
Song L, Song Z, Fry NJ, Conatser L, Llanga T, Mei H, Kafri T, Hirsch ML. Song L, et al. Hum Gene Ther. 2019 Nov;30(11):1336-1348. doi: 10.1089/hum.2019.071. Epub 2019 Sep 25. Hum Gene Ther. 2019. PMID: 31392914 Free PMC article. - 25 years of epidermal stem cell research.
Ghadially R. Ghadially R. J Invest Dermatol. 2012 Mar;132(3 Pt 2):797-810. doi: 10.1038/jid.2011.434. Epub 2011 Dec 29. J Invest Dermatol. 2012. PMID: 22205306 Free PMC article. Review. - Gene doping: Olympic genes for Olympic dreams.
Battery L, Solomon A, Gould D. Battery L, et al. J R Soc Med. 2011 Dec;104(12):494-500. doi: 10.1258/jrsm.2011.110240. J R Soc Med. 2011. PMID: 22179292 Free PMC article. No abstract available. - Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa.
Tolar J, Xia L, Riddle MJ, Lees CJ, Eide CR, McElmurry RT, Titeux M, Osborn MJ, Lund TC, Hovnanian A, Wagner JE, Blazar BR. Tolar J, et al. J Invest Dermatol. 2011 Apr;131(4):848-56. doi: 10.1038/jid.2010.346. Epub 2010 Dec 2. J Invest Dermatol. 2011. PMID: 21124339 Free PMC article.
Publication types
MeSH terms
LinkOut - more resources
Full Text Sources
Other Literature Sources
Medical