Genes and genetics

Hope for a cure hangs on research

Amid drastic proposed cuts to biomedical research, rare disease families like Hailey Adkisson’s fight for survival and hope. Without funding, science can’t “catch up” to help the patients who need it most.

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.

CRISPR epigenome editor offers potential gene therapies

Scientists from the University of California, Berkeley, created a system to modify the methylation patterns in neurons. They presented their findings at ASBMB 2025.

Using DNA barcodes to capture local biodiversity

Undergraduate at the University of California, Santa Barbara, leads citizen science initiative to engage the public in DNA barcoding to catalog local biodiversity, fostering community involvement in science.

Unraveling oncogenesis: What makes cancer tick?

Learn about the ASBMB 2025 symposium on oncogenic hubs: chromatin regulatory and transcriptional complexes in cancer.

A new kind of stem cell is revolutionizing regenerative medicine

Induced pluripotent stem cells are paving the way for personalized treatments to diabetes, vision loss and more. However, scientists still face hurdles such as strict regulations, scalability, cell longevity and immune rejection.