StemCell Therapy

What Do We Make?

Gene Tools makes Morpholino antisense oligos. Morpholino oligos bind to complementary RNA and get in the way of processes; they can knock down gene expression, modify RNA splicing or inhibit miRNA activity and maturation. Morpholinos are the premier knockdown tools used in developmental biology labs, the best RNA-blocking reagents for cells in culture and, as Vivo-Morpholinos, the most specific delivery-enhanced oligos available for other animal models. We are the sole commercial manufacturer selling research quantities of Morpholinos world-wide.

Morpholino oligos are short chains of about 25 Morpholino subunits. Each subunit is comprised of a nucleic acid base, a morpholine ring and a non-ionic phosphorodiamidate intersubunit linkage. Morpholinos do not degrade their RNA targets, but instead act via an RNAse H-independent steric blocking mechanism. With their requirement for greater complementarity with their target RNAs, Morpholinos are free of the widespread off-target expression modulation typical of knockdowns which rely on RISC or RNase-H activity. They are completely stable in cells and do not induce immune responses.

With their high mRNA binding affinity and exquisite specificity, Morpholinos yield reliable and predictable results. Depending on the oligo sequence selected, they either can block translation initiation in the cytosol (by targeting the 5' UTR through the first 25 bases of coding sequence), can modify pre-mRNA splicing in the nucleus (by targeting splice junctions or splice regulatory sites) or can inhibit miRNA maturation and activity (by targeting pri-miRNA or mature miRNA), as well as more exotic applications such as ribozyme inhibition, modifying poly-A tailing, blocking RNA translocation sequences or translational frameshifting. Morpholinos have been shown effective in animals, protists, plants and bacteria.

We are continually developing novel cytosolic delivery systems like our 'Endo-Porter' for cultured cells and our Vivo-Morpholinos for both cultures and in vivo delivery. With established delivery technologies it's easy to deliver Morpholinos into cultures, embryos or animals -- making Morpholinos the best tools for genetic studies and drug target validation programs.

What Sets Us Apart?

Morpholino oligos have excellent antisense properties compared to other gene knockdown systems. Microinjection or electroporation of Morpholino oligos into the embryos of frogs, zebrafish, chicks, sea urchins and other organisms successfully and specifically shuts down the expression of targeted genes, making Morpholinos an indispensable tool of developmental biologists. Morpholinos have also proven their versatility and efficacy in cultures of primary or immortal cells when delivered by Endo-Porter, electroporation or Vivo-Morpholinos. Usually, Vivo-Morpholinos are used to bring the specificity and efficacy of Morpholino oligos to experiments requiring systemic delivery in adult animals. The list of over 7500 publications using Morpholinos is growing daily and is maintained on-line in a browseable database.

Besides providing the best knockdown and splice modification tools, we also provide the best customer support available in the gene silencing industry. Our customer support team includes three Ph.D.-level scientists with hands-on Morpholino experience who are available to: 1) discuss your experiment design, 2) design your oligos for you, and 3) help you troubleshoot your experiments, all at no additional cost.

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This entry was posted on August 4, 2016 at 9:35 am and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |