In 2020, Jennifer Doudna, Ph.D., received the Nobel Prize in Chemistry, CRISPR-Cas9, a method for genome editing. Often referred to as “molecular scissors,” CRISPR cuts DNA at specific locations that ...
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 ...
In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...
Advanced engineered cell therapies require gene editing tools that are both precise and efficient. In recent years, CRISPR-Cas9 has emerged as the gold standard for editing genes with greater ...
A major CRISPR breakthrough came last May. Researchers at Penn Medicine and the Children’s Hospital of Philadelphia developed a personalized base-editing treatment in just 6 months. The treatment was ...
Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein ...
NEW ORLEANS, LA—An investigational CRISPR-Cas9 gene-editing therapy that targets angiopoietin-like protein 3 (ANGPTL3), which has a role in regulating lipid metabolism, appears to safely lower levels ...
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