In the world of biopharmaceutical innovation, 2024 will be remembered as CRISPR’s breakout year. In the spring, five patients with sickle cell disease began treatment with Casgevy, the first ...
Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid ...
A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...
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