UniQure 1 article

uniQure is a gene-therapy biotechnology company whose platform is centered on AAV gene therapy, with adeno-associated virus vector engineering describing a key vector, tissue or delivery feature. The company connects central nervous system gene delivery with HTT gene expression lowering; neurodegenerative genetic disease therapy supplies the vector, tissue or expression-control feature that shapes gene delivery. GenoMethods maps vectors, transgenes and individual gene therapies separately; the genetic-medicine profile of uniQure also includes stereotactic brain vector delivery.