Rare & Genetic Diseases
| 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.
Scientists harnessed the prokaryotic immune system to develop a groundbreaking gene editing technology that improves human health.
| 10+ min read
Researchers around the world are on a mission to bank biological samples from millions of people. So far, these efforts have disclosed the effects of arsenic exposure on the genome, supported development of early cancer diagnostics, and even revealed the ideal timing for COVID-19 boosters.
| 9 min read
In a journey that spanned the genomes of more than 150,000 Icelanders, archival samples, and a fetus in utero, researchers not only identified genetic variants missing from the population, but also proved that these missing mutations cause three rare genetic diseases.
| 2 min read
On a quest to bring new a new hemophilia gene therapy to the clinic, one research team leapt over hurdle after hurdle to produce enough viral therapeutics to treat a large number of patients.
| 3 min read
Wolfram Syndrome is a rare disease that causes a myriad of challenges, including deafness, diabetes, and depression. New research suggests that an agonist used to treat neurodegenerative disorders may help alleviate some of the symptoms.
| 2 min read
What do 23andMe and Snapchat have in common? They both collect very personal information about their users. Do the benefits outweigh the risks?
