Scientists create first humanized mouse model for rare genetic disease

Mice with a defective human gene responsible for a rare genetic disease called congenital adrenal hyperplasia, have been developed for the first time. The achievement, presented at the 25th European Congress of Endocrinology, may help to develop new therapies for people with the most common type of congenital adrenal hyperplasia.

source https://medicalxpress.com/news/2023-05-scientists-humanized-mouse-rare-genetic.html