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 ...

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

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

Achondroplasia, also known as short-limb dwarfism, is associated with neurological symptoms and complications due to narrowing of the skeletal structures surrounding the spinal cord.

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...