Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...

Worldwide, only a handful of patients are known to suffer from episodic ataxia type 6, a neurological disease that causes transient loss of muscle control. The cause lies in a mutation that changes a ...

Correlation does not equal causation. Every cancer cell has genetic mutations, but not all of those mutations necessarily ...

Scientists have corrected an extremely rare and life-threatening genetic disease of the liver in mouse models and human patient cells, using the gene-editing approach that served as the basis for the ...

Scientists have discovered a surprising mechanism by which the inherited genetic mutation known to cause Huntington's disease leads to the death of brain cells. The findings change the understanding ...

Drugs are potent weapons against viral pathogens. During the early stages of the COVID-19 pandemic, there were intensive efforts to discover and implement antiviral drugs that could treat SARS-CoV-2 ...

Gene therapy has the potential to dramatically benefit patients with genetic blood disorders, but a small study on participants in a clinical trial for sickle cell disease suggests that a key process ...

Revealing the limitations of cloning, researchers who repeatedly cloned mice for two decades have discovered that such serial duplication triggers grave genetic mutations that accumulate over the ...