Personalized gene editing corrects fatal infant metabolic disorder in world-first treatment

In a global first, scientists have used advanced base editing to repair a deadly gene mutation in a newborn, marking a major leap forward in the treatment of rare inherited metabolic diseases. Study: Personalized Gene Editing to Treat an Inborn Error of Metabolism. Image Credit: Shutterstock AI Generator / Shutterstock.com In a recent New England Journal … Read more

NIH researchers define topical steroid withdrawal diagnostic criteria

Researchers at the National Institutes of Health (NIH) have determined that dermatitis resulting from topical steroid withdrawal (TSW) is distinct from eczema and is caused by an excess of an essential chemical compound in the body. Scientists from NIH’s National Institute of Allergy and Infectious Diseases (NIAID) identified treatments that could be studied in clinical … Read more

APOBEC proteins linked to DNA repeat expansion in Huntington’s disease

People genetically susceptible to Huntington’s disease often see their movement, mood, and cognition decline slowly over time.  The cause is related to expansion of repeating DNA units, in which specific strings of genetic code-in this case, a series of cytosine-adenine-guanine nucleotides, or CAG, on one strand of the DNA and cytosine-thymine-guanine, or CTG, on the complementary … Read more