Crispr Therapeutics (CRSP) is downgraded to "Hold" as current valuation already reflects optimism for CTX310 Phase I results and pipeline progress. Intellia Therapeutics (NTLA) maintains a "Buy" ...

Developing a gene therapy typically takes years, but when Baby KJ was diagnosed with a deadly genetic condition, scientists had only months. What followed was a successful collaboration among ...

One of the most revolutionary tools in cutting-edge medicine is a molecular scalpel so precise that it can modify defective DNA and fix genetic diseases like sickle cell anemia, and chronic disorders ...

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 ...

Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT in 2021 after covering health and science at WIRED. You can reach ...

Scientists at The University of Texas at Austin have developed a revolutionary gene-editing method using bacterial retrons that can correct multiple disease-causing mutations at once. Unlike ...

A new study published in Nucleic Acids Research unveils crucial advances in the engineering of CRISPR-associated transposons (CASTs)—a class of natural, RNA-guided DNA integration systems. According ...

The rings of DNA inside mitochondria are inaccessible to these techniques, which means that precise edits to mitochondrial DNA (mtDNA) remain frustratingly out of reach. “Mitochondria missed the ...

A tiny genetic tweak can make pigs completely resistant to classical swine fever, a major problem for farmers around the world. The same gene edit should also make cattle and sheep resistant to ...

The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the CRISPR/Cas system, colloquially known as gene scissors. These can cut and ...

Faulty versions of the LMNA gene can cause a wide range of health problems, including heart muscle disease (dilated cardiomyopathy) and muscle weakness (muscular dystrophies). Many of these diseases ...