Today in @CellCellPress we report the use of prime editing to correct several mutations that cause alternating hemiplegia of childhood (AHC), a rare and devastating neurodevelopmental disorder, in patient-derived cells and in two mouse models. https://t.co/gS7NsGqjtr 1/10 https://t.co/IhnBJ8BC7S
STAT+: AlphaFold developer Google DeepMind to fund CASP as NIH funding falls short https://t.co/u7qu5HLWvb #biotech #news
A combination of FDA-approved #CancerDrugs has been shown to reverse brain changes and restore memory in an Alzheimer's mouse model, suggesting a new therapeutic approach for dementia. @ucsf @CellCellPress https://t.co/jrF9KFP0T6 https://t.co/vOZglaHV4V
Recent advances in biomedical research highlight the use of artificial intelligence and genome editing technologies to address complex diseases. Insilico Medicine employs AI to identify novel drug targets, with deuterated pirfenidone showing promising results in phase 2b trials for fibrosis. David Baker’s lab at the University of Washington uses AI to target intrinsically disordered proteins, which are difficult to drug and implicated in cancer, diabetes, and pain. Researchers have identified two FDA-approved cancer drugs that, in combination, reverse brain changes and restore memory in an Alzheimer's mouse model, suggesting potential new treatments for dementia. Additionally, prime genome editing (CRISPR 3.0) has been successfully used to correct mutations causing alternating hemiplegia of childhood (AHC) in patient-derived cells and mouse models, reducing symptoms and extending lifespan. This work, led by David Liu in collaboration with the Jackson Laboratory and the rare disease group RARE Hope, represents a significant step forward for treating rare genetic brain diseases. Meanwhile, Google DeepMind, the developer of AlphaFold, is stepping in to fund the Critical Assessment of Structure Prediction (CASP) competition as NIH funding declines, supporting continued innovation in protein structure prediction.