Abstract: Provided herein are compositions and methods for preparation of 5? end region-modified mRNAs. In particular, the instant disclosure relates to novel mRNA 5? end region motifs and sequence initiators therefore together with assays that are capable of measuring the aspects of the functionality of those motifs and sequence initiators. Further provided herein are compositions and methods of treating conditions related to coronary disease.

Abstract: Provided herein are compositions and methods for preparation of 5? end region-modified mRNAs. In particular, the instant disclosure relates to novel mRNA 5? end region motifs and sequence initiators therefore together with assays that are capable of measuring the aspects of the functionality of those motifs and sequence initiators. Further provided herein are compositions and methods of treating conditions related to coronary disease.

Abstract: Aspects of this invention, inter alia, relate to novel systems for targeting, editing or manipulating DNA in a cell, using novel synthetic RNA-guided nucleases (sRGNs). The sRGNs are derived from wildtype or parental small type II CRISPR Cas9 endonucleases.

Abstract: Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Abstract: Disclosed herein are novel gene editing systems capable of being delivered to a subject intravenously through a lipid nano particle pharmaceutical formulation and producing durable in vivo editing of a target gene, such as ANGPTL3, with high on-target gene editing efficiency, reduced or low off-target effect, and no germline editing. The gene editing systems comprise a chemically modified guide nucleic acid sequence with a spacer having a specified arrangement of deoxyribonucleotides and ribonucleotides. The novel gene editing systems comprise mRNA that encodes the gene editor proteins, which may include a modified nickase component. Methods of disease treatments using the gene editing systems are also disclosed.

Abstract: Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Abstract: Provided herein are compositions, methods of making the same, and methods for targeted delivery of therapeutic agents for modifying expression and function of target genes, e.g. proteins involved in lipid and cholesterol metabolism such as PCSK9.

Abstract: Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Abstract: Provided herein are methods of using a compound of Formula (I) or pharmaceutically acceptable salt thereof, for example, in the treatment of a mental health disorder or chronic inflammatory disease.

Abstract: Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Abstract: Provided herein are compositions and methods for preparation of 5? end region-modified mRNAs. In particular, the instant disclosure relates to novel mRNA 5? end region motifs and sequence initiators therefore together with assays that are capable of measuring the aspects of the functionality of those motifs and sequence initiators. Further provided herein are compositions and methods of treating conditions related to coronary disease.

Abstract: Provided herein are methods of using a compound of Formula (I) or pharmaceutically acceptable salt thereof, for example, in the treatment of a mental health disorder or chronic inflammatory disease.

Abstract: Described herein are methods of purifying polynucleotides, e.g., imRNA and oligonucleotides, e.g., probes, primers and siRNA, using monolithic columns with immobilized ligands coupled to the monolithic column. Also described are monolithic columns for purifying polynucleotides from a sample; and methods of preparing such columns.

Abstract: Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Abstract: Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.

Abstract: Described herein is method for purifying messenger RNA (mRNA) encoding a DNA endonuclease from a sample, the method comprising: (a) loading the sample comprising the mRNA onto a monolithic matrix comprising a poly(dT) or poly(U) nucleic acid molecule linked/coupled to the monolithic matrix under conditions allowing the mRNA to hybridize with the poly(dT) or poly(U) nucleic acid molecule; (b) eluting the mRNA from the monolith matrix after one or more contaminants have been separated from the bound mRNA; and (c) separating the mRNA from dsRNA by adsorption chromatography, thereby resulting in a purified mRNA solution.

Abstract: A biopsy and therapy device comprising: a needling unit for holding and inserting a biopsy needle; an imaging module comprising an ultrasound probe and an actuator for moving the probe in a reciprocal action; a first arcuate slide; a second arcuate slide in sliding engagement with the first arcuate slide and a linkage to which the first arcuate slide is mounted; said linkage arranged to move the first and second arcuate slides within a vertical plane; said needling unit mounted to said arcuate slide wherein the first and second arcuate slides are mounted perpendicular to each other so as to rotate the needling unit about respective first and second principal axes.

Abstract: An example device includes a mounting surface including a first material having a first coefficient of thermal expansion (CTE). The device includes an intermediate plate coupled to the mounting surface and comprising a second material having a second CTE. The device includes a ferrite core coupled to the intermediate plate and comprising a third material having a third CTE, wherein the second CTE of the intermediate plate is between the first CTE of the mounting surface and the third CTE of the ferrite core.

Abstract: Aspects of this invention, inter alia, relate to novel systems for targeting, editing or manipulating DNA in a cell, using novel synthetic RNA-guided nucleases (sRGNs). The sRGNs are derived from wildtype or parental small type II CRISPR Cas9 endonucleases.

Abstract: A biopsy and therapy device comprising: a needling unit for holding and inserting a biopsy needle; an imaging module comprising an ultrasound probe and an actuator for moving the probe in a reciprocal action; a first arcuate slide; a second arcuate slide in sliding engagement with the first arcuate slide and a linkage to which the first arcuate slide is mounted; said linkage arranged to move the first and second arcuate slides within a vertical plane; said needling unit mounted to said arcuate slide wherein the first and second arcuate slides are mounted perpendicular to each other so as to rotate the needling unit about respective first and second principal axes.