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Rare & Genetic Diseases
| 10+ min read
From reducing prion protein to fighting neuroinflammation, researchers try different techniques to combat deadly prion diseases.
| 5 min read
An understudied passageway linking the inner ear and the brain in mice offers a potential delivery route for restoring hearing in humans.
| 10+ min read
Researchers developed ingestible capsules that inject drugs into the stomach and small intestine, providing a novel oral delivery method for biologics.
| 9 min read
Currently, the only cure for alpha thalassemia is a bone marrow transplant, but the success of gene therapies for related conditions points to a potential new cure.
New technologies keep CRISPR/Cas9 in check and minimize off-target risks.
| 3 min read
James Fujimoto, Eric Swanson, and David Huang developed optical coherence tomography, which transformed ophthalmological practice and spurred new imaging applications.
| 8 min read
A one-time gene therapy aims to stop Huntington's disease at its source. But first, it has to survive clinical trials.
| 9 min read
This year’s Breakthrough Prizes honor advances in CAR T cancer therapies, cystic fibrosis, and Parkinson’s disease.
| 9 min read
Cell therapies derived from stem cells could one day prevent vision loss or even restore sight in retinal degenerative disease.
| 9 min read
Injecting healthy, exogenous mitochondria into the eye could combat mitochondrial dysfunction and cell death in Fuchs endothelial corneal dystrophy.
| 4 min read
Data from a highly resilient individual guided researchers to new potential therapeutic targets.
Key advances in antigen detection technology and antibody specificity allowed early at home pregnancy detection.
