Enzyme replacement therapy in ectodermal dysplasia

Posted by & filed under Part 22: CONNECTIVE TISSUE, Treatment.

Significant Correction of Disease after Postnatal Administration of Recombinant Ectodysplasin A in Canine X-Linked Ectodermal Dysplasia

Margret L. Casal, John R. Lewis, Elizabeth A. Mauldin, Aubry Tardivel, Karine Ingold,
Manuel Favre, Fabrice Paradies,* Ste´phane Demotz, Olivier Gaide, and Pascal Schneider

The American Journal of Human Genetics, volume 81 (2007)
This paper describes the application of enzyme replacement therapy for X-linked hypohidrotic ectodermal dysplasia in dogs. Ectodysplasin A fused to the Fc portion of human immunoglobulin G1 was administered to the dogs in th neonatal period. This treatment improved the teeth  weight gain, restored lacrimation, preventing eye infections and keratoconjuctivitis sicca, induced the formation of moderate numbers of functional sweat glands, and significantly improved mucociliary clearance in the respiratory tract that correlated with the marked decrease of pulmonary diseases in the treated dogs.

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Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator

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