Disease mechanisms and treatments

Before we’ve lost what we can’t rebuild: Hope for prion disease
Sonia Vallabh and Eric Minikel, a husband-and-wife team racing to cure prion disease, helped develop ION717, an antisense oligonucleotide treatment now in clinical trials. Their mission is personal — and just getting started.

Defeating deletions and duplications
Promising therapeutics for chromosome 15 rare neurodevelopmental disorders, including Angelman syndrome, Dup15q syndrome and Prader–Willi syndrome.

Using 'nature’s mistakes' as a window into Lafora disease
After years of heartbreak, Lafora disease families are fueling glycogen storage research breakthroughs, helping develop therapies that may treat not only Lafora but other related neurological disorders.

Cracking cancer’s code through functional connections
A machine learning–derived protein cofunction network is transforming how scientists understand and uncover relationships between proteins in cancer.

Neurobiology of stress and substance use
MOSAIC scholar and proud Latino, Bryan Cruz of Scripps Research Institute studies the neurochemical origins of PTSD-related alcohol use using a multidisciplinary approach.

Proteomic variation in heart tissues
By tracking protein changes in stem cell–derived heart cells, researchers from Cedars-Sinai uncovered surprising diversity — including a potential new cell type — that could reshape how we study and treat heart disease.

Calcium channel linked to cancer drug resistance
Researchers discover a protein associated with carboplatin-resistant retinoblastoma, suggesting this protein could be a promising therapeutic target. Read more about this recent Journal of Biological Chemistry paper.

Host fatty acids enhance dengue virus infectivity
Researchers in Germany find that viral replication depends on host enzymes that synthesize lipids, revealing potential metabolic targets for antiviral intervention. Read more about this recent Journal of Biological Chemistry paper.

Butter, olive oil, coconut oil — what to choose?
Depending on the chain length and origin of the fat, regular fat consumption changes the specific makeup of fats in bloodstream and affect mild to severe cholesterol patterns. Read about this recent Journal of Lipid Research study.