Intra-amniotic ER004 as a prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED)

• Unité de recherche: INNOSUISSE
• Numéro de projet: 120.370 IP-LS
• Titre du projet anglais: Intra-amniotic ER004 as a prenatal treatment of X-linked hypohidrotic ectodermal dysplasia (XLHED)

Mots-clé (Anglais)

Life sciences, Human medicine

Résumé des résultats (Abstract) (Anglais)

ER004 is the first therapy specifically addressing X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder caused by the lack of a functional ectodysplasin A1 (EDA1) protein. Intra-amniotic treatment with ER004 has the potential to switch off symptoms throughout patients? lives.