Project A04
In vitro and in vivo models of novel therapeutic approaches in congenital adrenal hyperplasia
The project A04 deals with the development of new treatments for Congenital Adrenal Hyperplasia (CAH) focusing on the therapeutic effect of MC2-receptor antagonists and pharmacological chaperones. For functional in vivo tests of novel therapeutic agents, a humanised CYP21A2-deficient mouse model has been developed and will be characterized. The existing paediatric and adult CAH databases will be combined into one longitudinal national database allowing for continuity, which will then represent an excellent database for translational CAH research.
Aims
(I) Develop improved antagonistic peptides suppressing the MC2R pathway
(II) Model and optimise pharmacological chaperones for rescuing CYP21A2 enzyme activity
(III) Characterise and utilise the CAH mouse model for in vivo tests of peptides and chaperones
(IV) Establish a national combined paediatric and adult CAH database
| Principal Investigators | Institution |
| Prof. Dr. Angela Hübner | LMU |
| Prof. Dr. Nicole Reisch | TUD |
| PD Dr. Katrin Köhler | TUD |
