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Hope for a cure hangs on research

Amid drastic proposed cuts to biomedical research, rare disease families like Hailey Adkisson鈥檚 fight for survival and hope. Without funding, science can鈥檛 鈥渃atch up鈥� to help the patients who need it most.

Before we鈥檝e lost what we can鈥檛 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 鈥� and just getting started.

Defeating deletions and duplications

Promising therapeutics for chromosome 15 rare neurodevelopmental disorders, including Angelman syndrome, Dup15q syndrome and Prader鈥揥illi syndrome.

Using 'nature鈥檚 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鈥檚 code through functional connections

A machine learning鈥揹erived 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鈥揹erived heart cells, researchers from Cedars-Sinai uncovered surprising diversity 鈥� including a potential new cell type 鈥� 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.