In July 2025, Novartis Pharmaceuticals conducted a Phase III multi-center, single dose (1.2 x 1014 vector genomes), ...

The discovery may explain why cutting edge CAR T-cell therapy has managed to revolutionized treatment for blood cancer but ...

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

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

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

Scientists at the University of Sydney have uncovered a malfunctioning version of the SOD1 protein that clumps inside brain ...

Fourteen million people worldwide suffer from enlarged hearts, or hypertrophic cardiomyopathy (HCM), a genetic disease that ...

A single mutation in the H5N1 strain of avian influenza that is infecting cows in the US could make it more likely to infect humans, warn researchers.