Gene Tools is the sole commercial manufacturer selling research quantities of Morpholino antisense oligos world-wide. To knockdown gene expression, redirect splicing or block miRNA maturation, Morpholino oligos are the premier tools of developmental biology labs, the best RNA-blocking reagents for cells in culture and, as Vivo-Morpholinos, the most specific knockdown technology 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 morpholine ring and a non-ionic phosphorodiamidate intersubunit linkage. Morpholinos act via an RNAse H-independent steric blocking mechanism. With their high mRNA binding affinity and exquisite specificity, Morpholinos yield reliable and predictable results. 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 can inhibit miRNA maturation and activity. 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 innate immune responses. We continually develop novel delivery systems like our 'Endo-Porter' for cultured cells and our Vivo-Morpholinos for in vivo delivery, making it easy to deliver Morpholinos into cultures or animals -- making Morpholinos the best tools for genetic studies and drug target validation programs. In recent years siRNA has supplanted phosphorothioate antisense as the method most investigators consider first for gene knockdowns in cultures or animals. However, as it was before with phosphorothioates, investigators have become frustrated with the experimental failures, the time and money required to screen sequences, the off-target expression modulation and the non-antisense effects associated with siRNA. Here you can read a 2007 paper discussing mechanisms, strengths and weaknesses of siRNA and phosphorothioate oligos versus Morpholino oligos.

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 and immortal cells when delivered by Endo-Porter or electroporation. Now, with Vivo-Morpholinos, the specificity and efficacy of Morpholino oligos can be brought to experiments in adult animals. The list of over 2100 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 scientifically active Ph.D.-level scientists 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. Gene Tools is a research-based company led by Dr. Jim Summerton (inventor of Morpholino oligos). While providing Morpholino oligos to researchers world-wide, Gene Tools continues to develop methods to improve in vivo delivery, in particular systemic in vivo delivery of therapeutically useful concentrations of Morpholinos into the cytosol of cells in adult organisms. Our research into in vivo delivery has led us to new molecules which are potentially useful for diagnostic and therapeutic applications in cancer; these are still in development.

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