Subject collection: Model Systems in Drug Discovery
- A mitochondrial therapeutic reverses visual decline in mouse models of diabetes
Summary: Visual decline in mouse models of diabetes is reversed, independently of treating other disease symptoms, by treatment with MTP-131, a water-soluble peptide that selectively targets cardiolipin and improves mitochondrial bioenergetics.
- Yeast as a system for modeling mitochondrial disease mechanisms and discovering therapies
Summary: In this Review, we discuss the use of budding yeast to understand mitochondrial diseases and help in the search for their treatments.
- Phenotype-driven chemical screening in zebrafish for compounds that inhibit collective cell migration identifies multiple pathways potentially involved in metastatic invasion
Summary: We have developed a phenotype-driven screen for identifying new inhibitors of collective cell migration and demonstrated the screen can successfully identify compounds active in vivo and potentially new pathways for targeting cancer metastasis.
- Cellular models and therapies for age-related macular degeneration
Summary: Here, we discuss the emerging cell-culture models and potential stem-cell-based therapies for AMD, a blinding disorder that affects millions of people worldwide.
- Sodium valproate increases the brain isoform of glycogen phosphorylase: looking for a compensation mechanism in McArdle disease using a mouse primary skeletal-muscle culture in vitro
Summary: Use of this in vitro model showed that sodium valproate (VPA) can reverse the muscle phenotype from a McArdle-like to a normal histological and biochemical profile.