Genetic editing holds promise to treat incurable diseases, but the most popular method—CRISPR—sometimes does more harm than good. A new study from University of California San Diego and Yale ...

Emerging gene editing platforms are revolutionizing precision medicine, enabling safe and effective genetic modifications for ...

Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

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 new scientific paper published last week by Dr. Dieter Egli and his genetics research team at Columbia University examines ...

Affecting an estimated 100,000 people globally, cystic fibrosis (CF) cases stem from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein. In the past several decades, ...

While researchers have long appreciated the enormous potential of genome editing to treat disease, applications of these technologies in humans have historically been limited by safety concerns. Even ...

Microbiome research has faced a hurdle from the get-go: the inability to edit the microbial genome in vivo. Until now, bacterial genomes had to be modified outside—and reintroduced into—the host ...

You may have seen it in the news recently: a baby in Pennsylvania with a rare genetic disorder was healed with a personalized treatment that repaired his specific genetic mutation. The treatment was ...

Genetic editing holds promise to treat incurable diseases, but the most popular method — CRISPR — sometimes does more harm than good. A new study from University of California San Diego and Yale ...