For decades, the dream of fixing harmful mutations in mitochondrial DNA felt out of reach. Scientists have long known these ...

The FDA has approved Zegfrovy (sunvozertinib) for locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations.

Replacing defective microglia can halt ALSP—a fatal neurodegenerative disease—in mice and humans, offering hope for broader brain therapies.

Microglia replacement therapy helps treat people with a rare genetic condition called ALSP, suggesting the approach could ...

Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) is a progressive neurological disease with an ...

DNA makes RNA, RNA makes protein, protein makes phenotype—was the guiding framework for understanding inheritance and disease ...

In a paper published last month, researchers at UT Southwestern Medical Center introduced lipid nanoparticles — hollow, fatty ...

Olaparib demonstrated activity in a phase 2 trial of patients who had advanced biliary tract cancer with homologous recombination repair (HRR) mutations. Results from this trial were presented at the ...

A groundbreaking study led by Prof. Bo Peng at Fudan University demonstrates that microglia replacement effectively halts the ...

The research looked specifically at blood stem cells, which live in the bone marrow and make different types of blood cells.

Rare genetic diseases are challenging for patients and their families—made all the more overwhelming because symptoms tend to ...

A new study from the University of California San Diego has uncovered a hidden cause of Alzheimer’s disease that may lead to ...