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In the study, published Monday (May 1) in Nature Biotechnology,  Luo and colleagues set their sights on two fusions genes they had previously found to be associated with prostate cancer and various forms of rapid and invasive cancer, including liver tumors. Using a modified CRISPR-Cas9 tool that creates a single- rather than double-stranded break in DNA, they targeted the chromosomal breakpoints that form these fusion genes and replaced fusion DNA with a gene encoding the enzyme HSV1-tk. This enzyme effectively kills tumor cells by converting the drug ganciclovir into its active form, which then blocks DNA synthesis and leads to cell death. (Ganciclovir is used to treat cytomegalovirus in humans.)

The Path to Myozyme Beginning in the 1960s and fueled by the biotechnology revolution in 1980s, researchers at academic centers around the world initiated work to identify a treatment for Pompe - including therapies that could replace the missing GAA enzyme in patients. Based on these early efforts, from 1998-2002 Genzyme worked to advance promising research involving four different drug candidates to treat Pompe:

LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).