Mutated DNA Restored to Normal in Gene Therapy AdvanceA single infusion successfully corrected a mutated gene, marking a significant advancement in gene therapy.This precision targeting could revolutionize treatment for various genetic diseases.
How Do We Defend Science?Threats to scientific funding endanger lives of patients with conditions like recessive dystrophic epidermolysis bullosa (RDEB).
'Future generations need cure' for brain disorderNeuroferritinopathy, a rare brain disorder affecting multiple generations of families, has led to a drug trial aimed at finding a treatment.
How Hypertrophic Cardiomyopathy Is DiagnosedHypertrophic cardiomyopathy (HCM) causes the heart muscle to thicken, leading to inefficiency and potential health complications.