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16 results for "genetic disease"
The FDA just unveiled a breakthrough framework that could bring life-saving gene therapies to thousands of children with rare diseases in a fraction of the time. For families who've watched their kids suffer while treatments stall, this changes everything.
Researchers have identified a tiny protein region that keeps hearts beating steadily throughout our lives. The breakthrough could lead to treatments for devastating inherited heart diseases that affect thousands of families.
Ty Sperle, 18, became the first person in the world cured of chronic granulomatous disease through groundbreaking gene editing treatment. His success opens the door for curing countless other rare genetic diseases affecting children everywhere.
Scientists have created a precise gene-editing tool that could offer permanent treatment for cystic fibrosis patients, potentially replacing daily medications that cost hundreds of thousands of dollars per year. The breakthrough could transform care for people living with this and other genetic diseases.
A 9½-month-old baby became the first person ever to receive a custom gene-editing treatment designed specifically for his unique mutation. The breakthrough offers hope for thousands of rare genetic diseases that were once considered unreachable.
Adults with a rare genetic disease called cystinosis no longer need dozens of pills each day after receiving a groundbreaking one-time gene therapy. Five years later, most patients show stable or improved health across multiple organs.
Scientists have developed a new gene editing approach that spreads healing changes from cell to cell, dramatically increasing its reach in the body. This breakthrough could transform treatment for countless genetic diseases that were previously too difficult to address.
Scientists are developing a breakthrough gene editing approach that spreads between cells like neighbors sharing flyers. This could treat far more diseases by reaching dramatically more cells in the body.
Google DeepMind just opened up its DNA-reading AI to help scientists worldwide crack the code of genetic diseases. Nearly 3,000 researchers are already using it to fight cancer, brain disorders, and infections.
Google's DeepMind just unveiled an AI that can read and predict the impact of mutations in the 98% of human DNA scientists barely understand. This breakthrough could accelerate the discovery of treatments for everything from rare genetic diseases to cancer.
Charlotte, 64, lost her sight at 17 due to a rare genetic disease. New AI-powered glasses are giving her the independence she hasn't had in decades.
Australian researchers developed an AI system that makes gene editing safer by protecting healthy genes during treatment. The breakthrough could transform how doctors treat genetic diseases like cystic fibrosis.
Melbourne researchers developed an AI tool that creates molecular "off switches" for CRISPR gene editing in just 8 weeks. The breakthrough could help make gene therapy treatments safer and more accessible for patients with genetic diseases.
Scientists at Carnegie Mellon University have discovered a breakthrough approach to target the root cause of myotonic dystrophy type 1, the most common form of adult muscular dystrophy. The precision therapy could finally offer hope to patients with a disease that currently has no effective treatment.
Scientists just proved that two harmful genetic mutations can actually restore normal function when combined, confirming a 60-year-old hypothesis. This breakthrough could transform how doctors diagnose and treat thousands of rare genetic diseases.
Scientists at Carnegie Mellon University have discovered a precise way to target toxic RNA that causes myotonic dystrophy type 1, the most common adult-onset muscular dystrophy affecting 1 in 2,300 people worldwide. The breakthrough could lead to treatments with fewer side effects for this currently incurable disease and other devastating genetic disorders.