Epidermolysis bullosa, challenges and novel therapies

Publish Year: 1398
نوع سند: مقاله کنفرانسی
زبان: English
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WTRMED06_011

تاریخ نمایه سازی: 5 بهمن 1398

Abstract:

Epidermolysis bullosa (EB) is a congenital and life threatening disease and a genetic disorder of connective tissue with a prevalence of 3500 patients in the United States of America until 2016. There are four major congenital types of disease including simplex, junctional, dystrophic and a newer type, kindler-be syndrome. Method: In order to obtain data for this review article we have searched databases and resources of PubMed and Scopus. The search keywords included Epidermolysis bullosa, EB, treatment, Gene therapy, hemi desmosome and Stem cell. First, we studied the titles and abstracts of articles, then we reviewed the full texts of more relevant studies and data extracted from them.Results: EB patients are at increased risk of death from their disease consequences before the third decent of them life. Gene correction and stem cell transplantation or combination of tissue engineering and gene therapy are modern suggested treatments for EB. This review spends to etiologies and mutations resulting in different types of the disease and efforts conducted in order to modify mutations, especially those affecting hemi desmosome structures in epidermal keratinocytes in different types of EB.Conclusion: In vitro gene therapy of keratinocyte stem cells and transplant them back to the skin as a novel suggested treatment option is expected to improve clinical features in EB patients. Application of viral vectors for gene therapy is restricted due to ethical issues and usage of non-viral vectors is more applicable for gene therapy in these patients. Authors conclude from previous researches that there is a proportional relation between numbers of each different subunits of hemi desmosome in its structure (disturbed in EB), which have been neglected and should be considered in future gene therapy researches.

Authors

Mohammad hossein Abbasi

PHD, student of Medicine (medical intern) – MPH student Iran university of medical sciences, Tehran, Iran