Gene therapy for recessive dystrophic epidermolysis bullosa.
Among the severe genetic disorders of the skin that are suitable for gene and cell therapy, most efforts have been made in the treatment of blistering diseases including dystrophic epidermolysis bullosa. This condition can be recessively or dominantly inherited, depending on the nature and position of the mutation or mutations in the gene encoding type VII collagen. At present, there is no specific treatment for recessive dystrophic epidermolysis bullosa, and gene and cell therapy approaches hold great promise. This article discusses the different gene therapy approaches that have been used for the treatment of this disease and the new perspectives that they open.
Titeux
Matthias
M
Institut National de la santé et de la recherche médicale, U563, Toulouse France.
Pendaries
Valérie
V
Hovnanian
Alain
A
D024143:Collagen Type VII / Q000235:genetics*; D016108:Epidermolysis Bullosa Dystrophica / Q000235:genetics* / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D006801:Humans
D024143:Collagen Type VII