mitoCELL:
Centralized Service Structure for Myoblast Cultures of Mitochondrial diseases
State of the art
The rare mitochondrial diseases addressed by the MitoNet consortium are markedly heterogeneous and may manifest as fatal multisystem disorders of infancy, severe neurological syndromes in adolescents and young adults or as milder oligosymptomatic variants in older adults. They often lead to chronic morbidity and increased mortality. The enormous progress that has been achieved in the last two decades in deciphering the molecular mechanisms of mitochondrial diseases has not led until now to major improvements of treatment, not to mention causal therapies. This is all the more unfortunate, as the primarily functional energy deficiency may well be amenable to energy-restoring therapies. Thus, the absence of therapeutic options is not caused by principal biological limitations, but rather by missing of suitable laboratory or cellular model systems to evaluate therapeutic or diagnostic options.
Aims of the project
mitoCELL aspires to be a central service structure within MITONET with the principal objective of making primary myoblast and fibroblast cultures of patients with mitochondrial disorders available to all scientists within the network. By supplying viable myoblasts/fibroblasts/DNA/RNA, MTCC may circumvent a major limitation in the translation of molecular technologies to individual clinical applications and will therefore help to improve diagnostic and therapeutic efforts in the care of patients with rare mitochondrial diseases.
Project management and contact addresses
Angela Abicht, PD Dr. med. (principal investigator)
Department of Neurology, Friedrich-Baur-Institut
University of München
Ziemssenstr. 1, 80336 München
Tel: +49-89-5160-7470
Fax: +49-89-5160-7402
E-mail: angela.abicht@med.uni-muenchen.de
Peter Schneiderat, Dr. med.
Department of Neurology, Friedrich-Baur-Institut
University of München
Ziemssenstr. 1, 80336 München
Tel: ++49-89-5160-7470
Fax: +49-89-5160-7402
E-mail: peter.schneiderat@med.uni-muenchen.de
