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Genetic and Rare Diseases Information Center (GARD)

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Ectodermal dysplasia

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Your Question

Is there any way of repairing or replacing the missing gene in individuals with ectodermal dysplasia?

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

Is gene therapy available for individuals with ectodermal dysplasia?

The current research for treatment options for ectodermal dysplasia involves protein therapy but not gene therapy. Protein therapy aims to replace the missing protein that is causing a medical condition. There is an ongoing protein therapy clinical trial that seeks to replace the missing protein in individuals with X-linked hypohidrotic ectodermal dysplasia (XLHED). The ectodysplasin- A1 protein (EDA-A1), which is involved in the formation and development of skin and teeth, is found naturally in healthy people. Patients with XLHED do not produce this protein. ED1200 is a man-made form of EDA-A1 that was found to improve the development of hair, teeth, skin and some glandular structures in animals.[1] The current clinical trial seeks to obtain data that will be useful for determining the appropriate dosing of ED1200 for babies born with XLHED. To view information about this clinical trial please click here.

The XLHED Network is a group for patients and familes afffected by hypohidrotic ectodermal dysplasia or XLHED. Members of the network receive updates on HED and XLHED research and upcoming clinical trials. To learn more about this network please visit the following link.
Last updated: 9/18/2012