Researchers have developed a groundbreaking method for inserting large genes into specific locations in human cells, potentially transforming the field of gene therapy and opening new avenues in medical research.
In a groundbreaking case, a 6-month-old baby boy named KJ was treated with a personalized CRISPR gene-editing therapy, correcting a dangerous mutation before it could threaten his life. This innovative approach promises hope for similar cases.
A baby diagnosed with a rare genetic disorder has shown remarkable improvement after receiving an experimental gene editing treatment tailored just for him. This groundbreaking therapy offers hope for others with similar conditions.
Emily Kramer-Golinkoff, a cystic fibrosis patient with a rare mutation, highlights the struggles of those left behind as gene therapies show promise for others. Her journey underscores the need for equitable treatments in rare genetic conditions.
