Therapies for inherited skin fragility disorders.
Inherited skin fragility comprises disorders characterized by mechanical induced blistering and erosions within the skin and mucosal membranes as a consequence of mutations in genes encoding proteins involved in intra-epidermal or dermal-epidermal adhesion. As the molecular pathology is largely known, it is a prototype group of disorders for which numerous experimental treatments have been developed. However, it became clear that single therapeutic strategies will not be able to address all molecular and clinical aspects. Significant progress has been achieved in gene, cell and protein therapies. Although the way towards clinical application seems obvious, major challenges must be addressed before these therapies become largely accessible. Until curative treatments will become available, alternative strategies which aim at increasing protein stability, amending apoptosis, inflammation and scarring may alleviate skin fragility and prevent or delay the onset of complications.
Department of Dermatology, Medical Center - University of Freiburg, Freiburg, Germany.
Cristina
C
Has
Dimitra
D
Kiritsi
D064987:Cell- and Tissue-Based Therapy; D004820:Epidermolysis Bullosa / Q000235:genetics* / Q000628:therapy*; D016326:Extracellular Matrix Proteins / Q000235:genetics / Q000627:therapeutic use; D015316:Genetic Therapy; D006801:Humans; D011994:Recombinant Proteins / Q000235:genetics / Q000627:therapeutic use
D016326:Extracellular Matrix Proteins; D011994:Recombinant Proteins
adhesion; collagen VII; epidermolysis bullosa; keratin; laminin