Application of the high-precision technique is part of a clinical trial to treat a genetic disease that can cause liver and ...
A pH- and ROS-responsive hydrogel carrying CRISPR-Cas9 silenced the TRPV4 calcium channel in rat discs, restoring autophagic ...
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 ...
Scientists can now edit genes in precise locations deep in the brain and body without the use of invasive surgery or implants ...
A personalized CRISPR treatment for a critically ill baby shows how editing, RNA manufacturing, and delivery are converging ...
In a new study published in Nature titled, “Custom CRISPR-Cas9 PAM variants via scalable engineering and machine learning,” researchers from Massachusetts General Hospital (MGH) and Harvard Medical ...
The gene editing tool known as CRISPR-Cas9 is changing what's possible for treating a wide range of diseases caused by genetic mutations. But so far, attempts to use the technology to address ...
Emerging gene-editing platforms are demonstrating that disease-causing mutations, aberrant gene expression, and even large-scale DNA insertions can be corrected without relying on error-prone DNA ...
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 ...
A new review maps how mutations in more than twenty genes weaken bone in osteogenesis imperfecta and surveys therapies from ...
Don't read too much into this year's lackluster performance. Near-term catalysts are firming up.
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