Genetic Disorders

Influencers Fundraising Prince Frederik of Luxembourg Memory Gene Therapy Machine Learning Epigenetics Quality of Life Stem Cell Research Gene Editing

Nicotinamide Riboside Improves Cardiovascular, Skin and Kidney Health in Werner Syndrome

This first-of-its-kind crossover trial underscores the therapeutic promise of restoring NAD+ levels to counter accelerated aging complications.

Personalized CRISPR Therapy Sends Baby KJ Home from CHOP

FDA Halts Rocket’s RP-A501 Gene Therapy for Danon Disease After Patient’s Death

Landmark Study Maps Rapidly Mutating DNA Regions Across Four Generations

Researchers Release AI-Driven Tool for Custom CRISPR-Cas9 Enzyme Design

Breakthrough Liver Organoids Advance Regenerative Medicine and Drug Testing

Influencer Hannah Campbell Mourns Death of 10-Month-Old Daughter From Rare Genetic Disorder

Nitisinone Identified as Promising Mosquito-Killing Tool in Malaria Fight

Prince Frederik of Luxembourg Remembered for Advocacy Amid Rare Disease Battle

Maternal X Chromosome Found to Accelerate Brain Aging in Female Mice