Gene therapy and protein therapy of ectodermal dysplasias: A perspective

Abstract

Ectodermal dysplasias (EDs) form a complex and heterogeneous group of diseases currently defined and classified according to their clinical symptoms. The characterization, for several EDs, of the molecular events underlying their development, not only challenges this classification but also opens the door to new therapeutic options such as gene or protein therapy. This article provides a concise overview of the most recent successes and failures of this new type of treatment and sets in perspective how the specificities of given EDs will influence their feasibility in the near future. It makes the case for the need of new classification of EDs that is based on our most recent knowledge of the molecular basis of these diseases. © 2009 Wiley-Liss, Inc.