9 results about "Morpholino Oligonucleotides" patented technology

Methods featuring phosphoryl guanidinium based backbone and morpholino with phosphoramidite chemistry result in neutral antisense oligonucleotides. The PGMO is composed of a morpholino backbone linked to a phosphoryl guanidinium internucleotide (PG) linkage. These oligonucleotides can be used to treat cancer, autoimmune diseases, and other rare diseases.

The present invention relates to 5′-morpholino amidite monomers as 5′-CE / 5′-tBu phosphoramidite morpholino monomers (2) and process chemistry for the efficient synthesis of N-Trityl or monomethoxytrityl (MMTr)-protected 5′-morpholino amidite monomers and their use in the synthesis of phosphorodiamidate morpholino oligonucleotides (PMO), thiophosphoramidate morpholino oligonucleotides (TMO) and their chimeras which can be used for antisense technology or in general oligonucleotides related research.

The utility model relates to a continuous flow preparation device of morpholino oligonucleotide, and belongs to the field of morpholino oligonucleotide preparation devices. A continuous flow preparation device of morpholino oligonucleotide comprises an automatic synthesizer, a heating device and a constant temperature device, a synthesis column is arranged in the automatic synthesizer, a liquid inlet pipe is connected between an inlet of the synthesis column and the automatic synthesizer, and a liquid outlet pipe is connected between an outlet of the synthesis column and the automatic synthesizer; the heating device covers the surface of the composite column; and the constant temperature device covers the outer wall of the liquid outlet pipe. During continuous flow preparation of morpholino oligonucleotides, the coupling reaction efficiency can be effectively improved, and the impurity content can be controlled.

The application discloses a synthesis method of phosphorodiamidate morpholino oligonucleotide, belongs to the technical field of solid-phase synthesis of phosphorodiamidate morpholino oligonucleotide, and specifically relates to the following steps: adding 9-fluorenylmethyloxycarbonyl-piperazine-succinic acid into Wang resin to mix and react to synthesize 9-fluorenylmethyloxycarbonyl-piperazine-succinic acid-Wang resin, then adding phosphoramidite monomers to perform coupling reaction after deprotection treatment, performing cap treatment after the coupling is completed, then repeatedly performing deprotection treatment, coupling reaction and cap treatment until the coupling of all phosphoramidite monomers is completed, then removing the protecting group of the last phosphoramidite monomer, and then performing cleavage treatment and deprotection treatment of a base protecting group to obtain a phosphorodiamidate morpholino oligonucleotide (PMO) reaction solution. The application provides a synthesis method of phosphorodiamidate morpholino oligonucleotide with high coupling efficiency, low impurity level, high yield and high purity.

Methods of making and using nucleotide repeat phosphorodiamidate morpholino oligonucleotides (PMO) are provided. Nucleotide repeat PMOs include dinucleotide repeat PMOs, trinucleotide repeat PMOs and tetranucleotide repeat PMOs.

Methods featuring phosphoryl guanidinium based backbone and morpholino with phosphoramidite chemistry result in neutral antisense oligonucleotides. The PGMO is composed of a morpholino backbone linked to a phosphoryl guanidinium internucleotide (PG) linkage. These oligonucleotides can be used to treat cancer, autoimmune diseases, and other rare diseases.

The present disclosure provides human-CD47-specific antisense morpholino oligonucleotides useful to reduce expression of CD47 protein, for instance in order to reduce genotoxicity secondary from chemotherapy or radiation. Methods of use of the morpholinos are also provided.

Use of antisense thiomorpholino oligonucleotides to treat, prevent, or ameliorate the progression of inflammatory skin diseases or conditions such as psoriasis. Specifically, thiomorpholino¬ containing ASOs that target pre-mRNAs associated with inflammatory skin diseases or conditions such as psoriasis.

Composition and methods for treating neurodegenerative diseases, such as Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD), and Angelman's Syndrome (AS), the compositions specifically including antisense oligonucleotides (ASOs) containing thiomorpholino nucleotides configured to inhibit ribosomal frameshifting of paternally expressed gene 10 (PEG 10) mRNA during translation, thereby inhibiting the formation of the long form gag-pol protein.