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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.

Morpholino oligos are short chains of about 25 Morpholino subunits. Each subunit is comprised of a nucleic acid base, a methylenemorpholine 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. They are completely stable in cells, uncut by nucleases. Requiring greater complementarity with their target RNAs to affect gene expression, Morpholinos perturb the expression of untargeted genes far less than the widespread off-target expression changes typical of knockdowns relying on RISC or RNase-H activity.

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.

Several FDA-approved drugs are Morpholino oligos (eteplirsen, casimersen, golodirsen and viltolarsen). Morpholinos from Gene Tools are used in preclinical drug discovery and sometimes combined into larger systems (e.g. nanoparticle vehicles, antibody conjugates, etc.). Gene Tools does not manufacture GMP oligos, but GMP syntheses are available from other companies (such as Ajinomoto Bio-Pharma Services).

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 and adult animals using our cytosolic delivery systems like 'Endo-Porter' for cultured cells and 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.

The list of over 12,000 publications using Morpholinos is growing daily and is maintained on-line in a browseable database. Scroll down on this home page for some very recent citations describing work with Morpholinos or Vivo-Morpholinos; we also post these as we find them on X (formerly Twitter) and on Mastodon with the hashtag #Morpholino. Linked through the Products and Applications tab atop this page you can find lists of citations for oligo target types or various organism groups (e.g. Morpholinos for Blocking MicroRNAs, Morpholinos for Modifying Poly-A Tailing, Applications in Bacteria, etc.).

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.