Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020.
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ABSTRACT:
SUBMITTER: Has C
PROVIDER: S-EPMC7264085 | biostudies-literature | 2020 Jun
REPOSITORIES: biostudies-literature
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Molecular Therapeutics in Development for Epidermolysis Bullosa: Update 2020.
Has Cristina C South Andrew A Uitto Jouni J
Molecular diagnosis & therapy 20200601 3
Epidermolysis bullosa (EB) is a group of rare genetic disorders for which significant progress has been achieved in the development of molecular therapies in the last few decades. Such therapies require knowledge of mutant genes and specific mutations, some of them being allele specific. A relatively large number of clinical trials are ongoing and ascertaining the clinical efficacy of gene, protein or cell therapies or of repurposed drugs, mainly in recessive dystrophic EB. It is expected that s ...[more]
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