Abstract: What is described is a method of synthesis of the compound of formula 1A, or a salt thereof, wherein R3 is a linear or branched alkene of 1, 2, 3, 4, 5 or 6 carbons; R4 and R5 are the same or different, each a hydrogen, or a linear or branched alkyl of 1, 2, 3, 4, 5 or 6 carbons; and L3 is a bond or an alkane of 1, 2, 3, 4, 5 or 6 carbons.

Abstract: The present invention discloses a method for preventing dsRNA bacterial solutions from being degraded by UV light and application thereof in control of silverleaf whiteflies which relates to technical field of pest control. The method for preventing dsRNA bacterial solutions from being degraded by UV light of present invention is to mix dsRNA bacterial solutions with nanomaterial solutions to prepare mixed solutions of nanomaterial as well as dsRNA bacterial solutions, wherein the nanomaterial is one of MON, Fe3O4, TiC and SiC. Nanomaterials are used to protect dsRNA bacterial solutions from being degraded by UV light, so that lethal effect of dsGawky on silverleaf whiteflies can achieve purpose of pest control. The method is easy to operate, low in cost, good in effectiveness as well as sensitivity, high in insecticidal efficiency, and has other advantages such as environmental friendliness as well as good application prospects.

Abstract: A drug for the prevention or treatment of sepsis is provided. The drug comprises an exosome containing a circRNA MOTOR, and a nucleotide sequence corresponding to the circRNA MOTOR is shown in SEQ ID NO: 1.

Abstract: The invention relates to mutant forms of CsgG. The invention also related to analyte detection and characterisation using CsgG.

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

Abstract: Provided herein are methods and compositions for synthesizing 5?Capped RNAs wherein the initiating capped oligonucleotide primers have the general form m7Gppp[N2?Ome]n[N]m wherein m7G is N7-methylated guanosine or any guanosine analog, N is any natural, modified or unnatural nucleoside, “n” can be any integer from 0 to 4 and “m” can be an integer from 1 to 9.

Abstract: Aspects of the disclosure relate to a nucleic acid comprising a heterologous nucleic acid insert flanked by interrupted self-complementary sequences, wherein one self-complementary sequence is interrupted by a cross-arm sequence forming two opposing, lengthwise-symmetric stem-loops, and wherein the other of the self-complementary sequences is interrupted by a truncated cross-arm sequence. Methods of delivering the nucleic acid to a cell are also provided.

Abstract: A method for investigating intra- and/or intermolecular interactions involving RNA is provided. The method includes a) synthesizing a RNA/DNA heteroduplex (RDH) comprising a RNA strand of interest paired to a DNA strand; b) binding a first end of the DNA strand and a corresponding first end of the RNA strand to a first element of a nanoscale manipulating device, and a second end of the DNA strand to a second element of the nanoscale manipulating device, leaving a second end of the RNA strand free; c) moving the first and second elements of the manipulating device apart from each other, stretching the DNA strand and causing the RNA strand to peel off the heteroduplex; and d) moving the first and second elements of the nanoscale manipulating device towards each other, allowing the DNA strand to relax and causing the RNA strand to bind again to it. Measurement of a force-displacement relationship during steps c) and d) provides information on intra- and/or intermolecular interactions involving the RNA strand.

Abstract: Provided herein are methods for the treatment of bladder cancer.

Abstract: Disclosed herein are Advanced RNA Targeting (ARNATAR) dsRNA compounds targeting angiotensinogen (AGT). Such compounds are useful in methods for reducing expression of AGT and for therapeutically treating RAAS associated diseases, disorders and/or conditions, or symptoms thereof in a subject.

Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by improved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

Abstract: The present disclosure provides nucleic acid constructs for the treatment of cancer, comprising a cancer-specific promoter and one or more therapeutic genes.

Abstract: The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by inproved processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof.

Abstract: Disclosed herein are compositions and pharmaceutical formulations that comprise a binding moiety conjugated to a polynucleic acid molecule and a polymer. Also described herein include methods for treating a cancer which utilize a composition or a pharmaceutical formulation comprising a binding moiety conjugated to a polynucleic acid molecule and a polymer.

Abstract: The present invention relates to a composition for treating and preventing degenerative brain diseases comprising a complex of vitamin C and an aptamer binding to the vitamin C as an active ingredient, and the composition of the present invention has improved and therapeutic effects in a Parkinson's disease experimental model and so the composition of the present invention can be used as medicines and health functional foods for patients with degenerative brain diseases including Parkinson's disease.

Abstract: Compositions and methods for treating enzyme-deficiency diseases are disclosed. Multidomain therapeutic proteins containing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The multidomain therapeutic proteins are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.

Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting DGAT2 expression, which may be useful for treating, preventing, or ameliorating a disease associated with DGAT2.

Abstract: Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.

Abstract: The invention relates to new in vitro methods for synthesising a polymer, particularly a polynucleotide molecule, having a pre-defined sequence of units such as nucleotides. For synthesising a polynucleotide molecule the methods involve a process of extending a polynucleotide synthesis molecule with a transfer nucleotide. The methods additionally involve repeating the extension process multiple times to iteratively extend the polynucleotide molecule with multiple transfer nucleotides to generate a new polynucleotide molecule having a pre-defined nucleotide sequence. The invention also relates to in vitro methods of joining multiple synthetic polynucleotides following synthesis to form larger synthetic polynucleotides, as well as devices and systems for performing the extension, synthesis and assembly methods of the invention.

Abstract: Provided herein are antisense agents for knocking down, inhibiting, or silencing expression of an HNF4?-P2 isoform mRNA in a cell or a patient. Also provided are methods of knocking down, inhibiting, or silencing expression of an HNF4?-P2 isoform mRNA in a cell or a patient and methods of treating a patient with liver disease, liver damage, liver inflammation, or liver failure, such as acute liver failure, ALD, AH, or ACLF.