Rare & Genetic Diseases
Advanced biomedical technologies deliver new possibilities for studying complex biological systems.
Available on Demand
Scientists tackle AAV gene therapy hurdles, from enhancing efficiency to managing immunogenicity to improve AAV gene therapy efficacy and safety.
| 4 min read
By analyzing the changing genomes of short- and long-lived rockfish, scientists identified a new set of genes that might alter lifespan.
| 10+ min read
Forty years after the Orphan Drug Act passed, researchers advance drug development for neglected rare conditions everywhere from the lab bench to backstage.
| 3 min read
Legislation now allows some drugs to skip animal testing, but it’s unclear when, if ever, this step will be gone for good.
| 6 min read
For cases where gene editing simply won’t work, biochemist Aseem Ansari is working on a new idea: changing how cells regulate genes.
| 10+ min read
George Church is at it again, this time using multiplex gene editing to create virus-proof cells, improve organ transplant success, and protect elephants.
| 7 min read
Researchers correcting the genetic causes of disease at the DNA or RNA level make their cases.
| 6 min read
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.
| 4 min read
Neuroscientists safely delivered a critical gene that improved vision for two patients with Leber congenital amaurosis.
| 6 min read
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.
