Gene therapy and protein therapy of ectodermal dysplasias: a perspective.
Details
Serval ID
serval:BIB_1C3C9EE587E2
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Gene therapy and protein therapy of ectodermal dysplasias: a perspective.
Journal
American journal of medical genetics. Part A
ISSN
1552-4833 (Electronic)
ISSN-L
1552-4825
Publication state
Published
Issued date
09/2009
Peer-reviewed
Oui
Volume
149A
Number
9
Pages
2042-2044
Language
english
Notes
Publication types: Journal Article
Publication Status: ppublish
Publication Status: ppublish
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.
Keywords
Ectodermal Dysplasia/diagnosis, Ectodermal Dysplasia/genetics, Ectodermal Dysplasia/therapy, Ectodysplasins/genetics, Ectodysplasins/therapeutic use, Genetic Therapy, Humans, Proteins/genetics, Proteins/therapeutic use
Pubmed
Open Access
Yes
Create date
02/11/2017 11:04
Last modification date
20/08/2019 12:52