Gene therapy advances shine the spotlight on epidermolysis bullosa, bringing hope to patients.
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Characterization of wound microbes in epidermolysis bullosa: A focus on Pseudomonas aeruginosa.
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Characterization of wound microbes in epidermolysis bullosa: A focus on Pseudomonas aeruginosa.
26 June 2023

Mol Ther. 2023 Jun 26:S1525-0016(23)00313-1. doi: 10.1016/j.ymthe.2023.06.002. 

Gene therapy advances shine the spotlight on epidermolysis bullosa, bringing hope to patients.

Authors: Koller U.


Abstract:

Recent breakthroughs in gene therapy continue to transform the landscape of therapies for epidermolysis bullosa (EB), offering encouraging outcomes for patients suffering from this severe blistering skin disease. Two different therapeutic strategies that have already reached the clinic share the common goal of restoring the missing or dysfunctional protein via gene replacement. Notably, while ex vivo approaches have shown success in the past, a ground-breaking in vivo gene therapy product has recently gained approval from the US Food and Drug Administration, marking a significant milestone for EB therapy.

Download (a pagamento): https://pubmed.ncbi.nlm.nih.gov/37369206/