Gene Tools, LLC designs and manufactures Morpholino antisense oligos. Morpholino oligos bind to complementary RNA and prevent binding of other macromolecules; they can knock down gene expression, modify RNA splicing and inhibit miRNA activity and maturation, among other applications. Morpholinos are used in embryos, in cell culture and, as Vivo-Morpholinos, in adult animal models.
Morpholinos have been approved by the FDA for human use. Eteplirsen, golodirsen, casimersen and viltolarsen are being sold and administered to people with Duchenne muscular dystrophy. Morpholinos from Gene Tools are research-grade, not GMP.
Our scientists developed novel cytosolic delivery systems like 'Endo-Porter' for cultured cells and ‘Vivo-Morpholinos’ for both cultures and in vivo delivery. Established delivery technologies can deliver Morpholinos into cultures, embryos and animals making Morpholinos the best tools for genetic studies and drug target validation programs.
Backed by Ph.D.-level customer support, Gene Tools sells research quantities of Morpholinos world-wide.