Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon ...
What if you could flip a genetic switch to silence a gene, then turn it back on with a simple drug? For researchers, gene-switch tools offer that kind of control—and a new system called Cyclone may ...