Abdominal Area, Kidneys & Pelvis
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Research Center On Rare kidney Diseases >>RECORD<<

Applicant: Prof. Dr. Mario Schiffer, Prof. Dr. Michael Wiesener
Institution: Universitätsklinikum Erlangen, Medizinische Klinik 4
EKFS funding line: Else Kröner Research School
Funding line:
Else Kröner Research Schools
Image: RECORD participants

In the field of rare diseases, it is important to develop innovative technologies to investigate the potential benefits of targeted drugs and to analyze the impact of possible gene therapy. Particularly in the field of rare diseases, animal models are very often not available to investigate the acute and long-term consequences of the correction of congenital or acquired genetic defects in body cells. For rare kidney diseases, both tissue culture and animal models are essential to establish basic laws on dose discovery, vector targeting, pharmacokinetics, toxicology and biomarkers for therapy monitoring and to develop drug investigations and tests of possible genetic targeting strategies in patients' own in vitro systems and humanized animal models.

Here you can find further information.