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SUMMARY:Drug discovery of neurological mitochondrial diseases with patient
 -specific brain organoids - Professor Dr. Alessandro Prigione
DTSTART:20240605T140000Z
DTEND:20240605T150000Z
UID:TALK216961@talks.cam.ac.uk
CONTACT:Lisa Arnold
DESCRIPTION:Energy metabolism is essential for providing the energy necess
 ary to ensure proper cellular function. Mutations in genes regulating this
  process lead to inherited metabolic disorders that can particularly affec
 t tissues with high energy demands like the brain. The limited access to p
 atient neural tissue and the difficulty to manipulate mitochondrial DNA co
 mplicates the development of transgenic animal models and cellular models\
 , which are needed for treatment dis-covery. \nIn this talk\, I will summa
 rize our efforts in using patient-derived and engineered induced pluripote
 nt stem cells (iPSCs) to study mitochondrial neurological diseases. I will
  show examples of this approach in the context of Huntington´s disease (H
 D) and Leigh syndrome (LS)\, which is the most frequent and most severe mi
 tochondrial disease affecting 1/40\,000 newborns. We show that neuronal cu
 ltures and brain organoids derived from HD and LS-specific iPSCs can be us
 ed as model systems to investigate the neuropathological mechanisms and to
  carry out phenotypic compound screenings. Our data pave the way to the id
 entification of disease-modifying therapies for currently incurable neurol
 ogical and mitochondrial disorders.\n
LOCATION:MRC MBU\, Level 7 Lecture Theatre\, The Keith Peters Building\, C
 B2 0XY
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