Mitochondrial Medicine BMBF e:BIO Junior Research Group
Induced pluripotent stem cells (iPSCs), generated through reprogramming of somatic cells back into an embryonic-like pluripotent state, hold great promises for biomedical research. The fundamental discovery that the cellular identity is plastic and amenable to be directed into different epigenetic states highlights the importance of cellular adaptations to novel environments and functional tasks. However, the nuclear-centric view of cell fate is no longer sufficient to fully describe these rapid and dramatic restructurings.
Our work focuses on the contributing role of mitochondria and energy metabolism in cellular plasticity and neural fate induction. We wish to build an iPSC-based approach for advancing the understanding and treatment of debilitating genetic brain disorders due to mitochondrial impairment.
The iPSC technology might lead to a novel paradigm in brain drug development of mitochondrial disorders, allowing the generation of live neural cells capable of recapitulating the genotype/metabotype of the affected tissues within the patients. Phenotypic preclinical compound screenings conducted on such patient-specific material may enable the identification of disease-modifying treatment strategies.
June 29, 2018 at 12 p.m. : Live Streaming Webinar on w/ Alessandro Prigione, Heather Christofk & Michael Teitell
The new reviews from our lab:
JMB Career Advancement Initiative:
BMBF newsletter on Rare Disease Day (Feb. 28, 2018) features our work:
Best Scientific Images Contest: 2nd place for
Phd Publication Price: 2nd place for
Agilent Seahorse, webinar
Thermo Fisher Scientific, 24 Hours of Stem Cells, webinar
Stem Cells concise review published:
MDC INSIGHTS featuring our research:
Our manuscript on mitochondrial disease drug discovery is out in Cell Stem Cell:
- Press release from the MDC:
- From the Mitochondrial Disease News:
Cell Stem Cell Preview published:
Our Special Issue in Seminars in Cell & Developmental Biologyis out!
Mitochondria are membrane-bound organelles acting as the power plants of the cell, because they generate most of the cell's supply of adenosine triphosphate (ATP). They have their own genome and also divide independently of the cell in which they reside. In addition to their bioenergetic role, mitochondria are involved in a number of cellular functions, including calcium and redox homeostasis signaling, cellular differentiation, apoptosis, as well as in the maintenance of cell cycle and growth.
Mitochondrial impairment has been implicated in several human diseases, such as Leigh syndrome, Parkinson’s disease, Huntington’s disease, Amyotrophic lateral sclerosis, and Autism. Mitochondrial disorders due to mutations in the mitochondrial DNA affect 1/5000 newborns. The nervous system is usually most affected, highlighting the dependence of neurons on mitochondrial functionality. No therapy or treatments currently exist, making mitochondrial diseases a significant burden for society.
Induced pluripotent stem cells (iPSCs) are generated by reprogramming adult somatic cells through forced expression of stem cell-specific transcription factors or related small molecules. iPSCs hold great promises in biomedical applications. Because they can propagate indefinitely, as well as give rise to every other cell type in the body, they may be employed for replacing defined tissues lost to damage or disease. Moreover, they can be used for model complex human disorders “in a dish”. Finally, iPSC derivatives may represent innovative cellular systems for the development of phenotype-driven drug discovery.