Usher syndrome is a genetic condition that causes deafness and sight loss. It can be caused by loss-of-function mutations in several different genes; the 1F subtype is caused by a lack of a functional copy of the gene PCDH15.
Similarly to the gene therapy for Duchenne muscular dystrophy that we reported on in BioNews 1196, PCDH15 is too long to fit inside a virus vector to be delivered into cells. US researchers have now engineered a shortened, but fully functional version of the gene, which they hope can be used to create a gene therapy for Usher syndrome, according to the Medicine Maker.
