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Rare & Genetic Diseases
| 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.
| 5 min read
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.
| 4 min read
Susan Kovats explores the role of sex hormones in autoimmunity to guide the development of therapeutics for lupus, Sjögren’s syndrome, and other diseases that preferentially affect women.
| 8 min read
Seeking alternatives to pharmacological approaches, researchers apply thermal, electrical, and mechanical stimuli to change nerve signaling and treat pain.
| 3 min read
Researchers are harnessing the power of silk to develop a shelf- and digestive tract-stable enzyme treatment for phenylketonuria.
| 3 min read
Researchers designed a lipid nanoparticle that sticks to bone minerals, increasing mRNA delivery and therapeutic protein expression in the bone.
| 5 min read
Samira Kiani combines her passion for art and design with synthetic biology to create safer CRISPR-based therapeutics for the future.
