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 ...
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 ...
Development of bespoke therapies has been a driving goal in medical research. This year, the announcement of the first patient treated with personalized CRISPR therapy, Baby KJ, highlights this ...
Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic diseases. By ...
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 ...
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, ...
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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