Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...
When the gene editing tool CRISPR-Cas9 rocketed to fame more than a decade ago, it transformed biotechnology. Faster, cheaper, and safer than previous methods, the tool helped scientists gain insight ...
CRISPR has the power to correct genetic mutations, but current delivery methods are either unsafe or inefficient, keeping the technology from reaching its full medical potential. With the power to ...
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 ...
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 ...
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 ...
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