The ethics of using safe gene therapies to improve the health and cognition of Down syndrome children and adults.

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

Intellia Therapeutics said its Crispr-based treatment for hereditary angioedema met its goals in a Phase 3 trial, marking a ...

CRISPR functions as a programmable genetic memory system derived from bacterial immune defenses against viral infections. Guide RNA allows for rapid multiplexed targeting compared to older ...

Aurora Therapeutics, cofounded by Nobel Prize–winning scientist Jennifer Doudna, plans to use gene editing and a new FDA regulatory pathway to commercialize treatments for rare diseases. Last February ...

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" ...

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 ...

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 ...

Like the human immune system, bacteria learn from past infections. CRISPR sequences—short snippets of DNA from previous viruses—guide destructive enzymes towards invading bacteriophages that express ...

A single infusion of an experimental gene-editing drug appears safe and effective for cutting cholesterol, possibly for life, according to a small early study released Saturday. The study, which ...