Scientists uncover genetic cause of rare autoimmune disorder
Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED) is a life-threatening autoimmune disease that affects ...
Helsinki4 年
Department of Dermatology and Allergology
As regards autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED), we have begun repairing the gene mutation with the CRISPR/Cas9 method in patients’ induced pluripotent stem cells ...
The Scientist1 年
The Future of Gene Therapy for a Rare Pediatric Autoimmune Disease
Bacchetta’s primary focus has been the rare autoimmune disorder IPEX syndrome, short for immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome. In this disorder, a mutation in the ...