RFR-PEL

The repeated clinical trial failures of Alzheimer’s disease therapies based on clearing beta-amyloid have been discouraging, but new research strategies and capabilities are providing renewed hope.

Consider this scenario: Jayne is 50 years old and has experienced progressing neurological symptoms over the past two years with no diagnosis. She recently had exome testing which show no genetic variants that explain her symptoms, but identifies a pathogenic variant (mutation) in MSH6.

A research team led by Albert Cheng developed a new method, CASFx, that leverages CRISPR technology to alter messenger RNA splicing and precisely control protein isoform production.

The traditional approach to genetic testing for those of Ashkenazi Jewish ancestry is under debate, with some experts calling for testing all Ashkenazi Jewish individuals and others advocating for further research to inform any changes in testing criteria.