Rare & Genetic Diseases

Scientists tackle AAV gene therapy hurdles, from enhancing efficiency to managing immunogenicity  to improve AAV gene therapy efficacy and safety.

By analyzing the changing genomes of short- and long-lived rockfish, scientists identified a new set of genes that might alter lifespan.

Forty years after the Orphan Drug Act passed, researchers advance drug development for neglected rare conditions everywhere from the lab bench to backstage.

Legislation now allows some drugs to skip animal testing, but it’s unclear when, if ever, this step will be gone for good.

For cases where gene editing simply won’t work, biochemist Aseem Ansari is working on a new idea: changing how cells regulate genes.

George Church is at it again, this time using multiplex gene editing to create virus-proof cells, improve organ transplant success, and protect elephants.

Researchers correcting the genetic causes of disease at the DNA or RNA level make their cases.

Kan Cao studies the rare aging disorder progeria to find a cure, and she’s ready to solve the mysteries of healthy human aging along the way.

The early steps of viral vector production influence cell and gene therapy success.

Neuroscientists safely delivered a critical gene that improved vision for two patients with Leber congenital amaurosis.

With diseases like cancer and kidney disease on the rise in Africa, Segun Fatumo and his team are sequencing the genomes of 100,000 Nigerians to understand why.

Researchers discovered that copper induces cytotoxicity through a distinct pathway termed cuproptosis. This insight guides the use of copper-shuttling drugs to treat copper regulation disorders and opens up new therapeutic applications for cancer.