Researchers used genome-wide CRISPR screens to identify genetic diseases responsive to vitamins B2 and B3. In mice lacking ...

RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...

By the 1890s, just as the medical world was beginning to understand that invisible microbes caused infectious diseases, the idea that a missing ...

A hallmark of Parkinson's disease is the buildup of Lewy bodies—misfolded clumps of the protein known as alpha-synuclein. Long before Lewy bodies form, alpha-synuclein can interfere with neurons' ...

When the Chernobyl power plant explosion scattered ionizing radiation all over Europe, the damage it dealt lasted much longer than the initial blast. Researchers sequenced the genomes of Chernobyl ...

PITTSBURGH--(BUSINESS WIRE)--#autism--The SCN2A Foundation today announced a research collaboration with Unravel Biosciences, Inc., an AI-enabled therapeutics company established to advance drugs for ...

A study led by Aaron Hobbs, Ph.D., and Rachel Burge, Ph.D., at MUSC Hollings Cancer Center, reveals why a specific gene mutation behaves differently from other variants. The study, published in Cancer ...

This study offers important insight into the pathogenic basis of intragenic frameshift deletions in the carboxy-terminal domain of MECP2, which account for some Rett syndrome cases, yet similar ...

Researchers discovered that a tiny structural feature of the enzyme GPX4 helps keep neurons safe. A rare mutation removes this protection, allowing harmful molecules to damage cell membranes and ...

Tatiana Schlossberg, the youngest granddaughter of President John F. Kennedy, recently revealed she was diagnosed with terminal acute myeloid leukemia (AML) due to a rare genetic anomaly. In a ...