1 in 12 people has an orphan disease. 95% of orphan diseases have no treatment.
Each disease is caused
by a spectrum of genetic mutations.
Perlstein Lab studies each mutation
in model organisms to produce precision medicine.
Leave no mutation behind
PLab is the first Public Benefit Corporation accelerating precision drug discovery for 5,000+ rare genetic diseases.
At PLab we’re always on the lookout for new publications describing simple animal models of rare genetic diseases. The more non-obvious the model/disease fit appears at first blush, the more exciting are any results that confirm therapeutic relevance in humans — or more accurately in human cells. Last week, a UK-based cystic fibrosis (CF) patient and rare disease advocate […]