273 results about "Messenger RNA" patented technology

Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.

Provided are compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression. Such compositions and methods include the use of modified messenger RNAs, and are useful for production of proteins.

A method for enrichment of natural antisense mRNA which involves hybridization of cDNA obtained from sense RNA with cDNA obtained from antisense RNA, followed by DNA polymerase treatment of the sense-antisense hybrid DNA molecule. A natural antisense library can be generated by cloning of sense-antisense hybrid DNA molecules in a vector.

The present invention provides compositions comprising mRNA molecules encapsulated within lipid particles. The lipid particles comprise a cationic lipid, a non-cationic lipid, and an mRNA molecule that is encapsulated within the lipid particle. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease. The invention also provides cationic lipids that are useful for preparing the compositions of the invention.

The present invention is directed to a synthetic nucleic acid sequence which encodes a protein wherein at least one non-common codon or less-common codon is replaced by a common codon. The synthetic nucleic acid sequence can include a continuous stretch of at least 90 codons all of which are common codons.

The present invention provides methods and compositions for generating novel nucleic acid molecules through targeted spliceosomal mediated RNA trans-splicing. The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a SERPINA1 target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention include those genetically engineered to interact with SERPINA1 target pre-mRNA so as to result in correction of SERPINA1 genetic defects responsible for AAT deficiency. The PTMs of the invention may also comprise sequences that are processed out of the PTM to yield duplex siRNA molecules directed specifically to mutant SERPIN A1 mRNAs. Such duplexed siRNAs are designed to reduce the accumulation of toxic AAT protein in liver cells. The methods and compositions of the present invention can be used in gene therapy for correction of SERPINA1 disorders such as AAT deficiency.

A method for enrichment of natural antisense mRNA which involves hybridization of cDNA obtained from sense RNA with cDNA obtained from antisense RNA, followed by DNA polymerase treatment of the sense-antisense hybrid DNA molecule. A natural antisense library can be generated by cloning of sense-antisense hybrid DNA molecules in a vector.

The present invention provides compositions and methods for inhibiting expression of a target gene in a cell. The process comprises introduction of double-stranded tripartite RNAi constructs into the cells and reducing the expression of the corresponding messenger RNA in the cells. The constructs, which may be packaged in or delivered as sequestered RNAi constructs, differ from the canonical siRNA in that they comprise a tripartite structure which follows the general formula of having (1) an RNAi core (either native or abbreviated), (2) one or more terminal moieties attached to the RNAi core and optionally (3) a linker between the RNAi core and the terminal moiety. Once packaged into sequestration vehicles, the constructs are activated for gene regulation by the application of certain forms of energy

The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a mRNA solution and a lipid solution, wherein the mRNA solution and / or the lipid solution are at a pre-determined temperature greater than ambient temperature.

The present invention is in the fields of molecular and cellular biology. The invention is generally related to reverse transcriptase enzymes and methods for the reverse transcription of nucleic acid molecules, especially messenger RNA molecules. Specifically, the invention relates to reverse transcriptase enzymes which have been mutated or modified to increase thermostability, decrease terminal deoxynucleotidyl transferase activity, and / or increase fidelity, and to methods of producing, amplifying or sequencing nucleic acid molecules (particularly cDNA molecules) using these reverse transcriptase enzymes or compositions. The invention also relates to nucleic acid molecules produced by these methods and to the use of such nucleic acid molecules to produce desired polypeptides. The invention also concerns kits comprising such enzymes or compositions.

A method of treating a disease resulting from the expression of a harmful gene is described. The method includes the step of administering a therapeutically effective amount of a pharmaceutical composition having at least one double stranded oligonucleotide including two complementary oligonucleotide sequences forming a hybrid. Each oligonucleotide sequence comprises at one of their 3′ or 5′ ends one to five unpaired nucleotides forming single-strand ends extending beyond the hybrid. One of the oligonucleotide sequences is substantially complementary to a target sequence belonging to a DNA or messenger RNA molecule of a gene coding a mutated or nonmutated androgen receptor.

The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly for mRNA synthesized in vitro. In some embodiments, the methods comprise chromatographic methods of quantifying capping efficiency and methylation status of the caps.

The present invention is directed to a synthetic nucleic acid sequence which encodes a protein wherein at least one non-common codon or less-common codon is replaced by a common codon. The synthetic nucleic acid sequence can include a continuous stretch of at least 90 codons all of which are common codons.

The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized mRNA to a denaturing condition, and purifying the mRNA from the impure preparation from step (a) by tangential flow filtration, wherein the mRNA purified from step (b) is substantially free of prematurely aborted RNA sequences and / or enzyme reagents used in in vitro synthesis.

The present disclosure relates generally to novel methods and compositions for using engineered reprogramming factor(s) for the creation of induced pluripotent stem cells (iPSCs) through a kinetically controlled process. Specifically, this disclosure relates to establishing combinations of reprogramming factors, including fusions between conventional reprogramming factors with transactivation domains, optimized for reprogramming various types of cells. More specifically, the exemplary methods disclosed herein can be used for creating induced pluripotent stem cells from various mammalian cell types, including human fibroblasts. Exemplary methods of feeder-free derivation of human induced pluripotent stem cells using synthetic messenger RNA are also disclosed.

The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA.

The present invention provides compositions comprising mRNA molecules encapsulated within lipid particles. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease. The invention also provided cationic lipids that are useful for preparing the compositions of the invention.

The present invention provides compositions comprising mRNA molecules encapsulated within lipid particles. The compositions are useful, for example, to introduce the mRNA molecules into a human subject where they are translated to produce a polypeptide that functions to ameliorate one or more symptoms of a disease. The invention also provided cationic lipids that are useful for preparing the compositions of the invention.

The invention provides a method for enriching target gene fragments. The method comprises the following steps: (1) obtaining a partial or whole-length DNA (deoxyribonucleic acid) sample library containing a target gene; (2) obtaining a DNA probe library capable of hybridizing with the target gene; (3) hybridizing the DNA probe library and the DNA sample library; (4) separating a hybrid product obtained in the step (3), and releasing the target gene fragments subjected to hybridizing and enriching. According to the enrichment method, the invention further provides a method for detecting the gene structure mutation of the target gene. The method for detecting the gene structure mutation of the target gene comprises the following steps: (1) enriching the target gene fragments according to the method; (2) detecting the structure mutation of the target gene. The target gene fragments enriched by using the method can be used for next-generation sequencing technique to detect the gene structure mutation and comprise single-alkali base mutation, mRNA (Messenger RNA) absence or increase, mRNA structure transversion and mRNA splicing change.

Hepatitis C virus (HCV) is a major cause of chronic liver disease and affects over 270 million individuals worldwide. The HCV genome is a single-stranded RNA that functions as both a messenger RNA and replication template, making it an attractive target for the study of RNA interference. Double-stranded short interfering RNA (siRNA) molecules designed to target the HCV genome are disclosed herein.

The present invention relates to the cloning of human pro-protein converting enzyme 5 (PC5) CDNA isolated from human adrenal gland messenger RNA. Additionally, this invention relates to a method for reducing restenosis occurring at an injured vascular site comprising delivering to the injured site an antisense nucleic acid to suppress the expression of human PC5.

Expression of a messenger RNA which encodes a protein which is expressed by differentiated cells, notably the cells of the skin or appendages thereof, is inhibited by topically administering to said differentiated cells a thus effective amount of at least one double-stranded RNA oligonucleotide, formulated into a topically applicable, physiologically acceptable medium therefor; such regime or regimen is useful for treating a variety of afflictions or conditions, e.g., combating the signs of skin aging, stimulating hair growth or retarding loss thereof, inhibiting cellular proliferation and / or differentiation, etc.

A system and method predict alternative splicing transcripts using DNA chip expression data as a primary data source. The system and method may perform prediction of alternative splicing of pre-messenger RNA that may be used, for example, for regulating eukaryotic gene expression.

The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, a step of centrifuging an mRNA suspension in a centrifuge comprising a porous substrate at a speed sufficient to remove process contaminants and to precipitate purified mRNA composition onto the porous substrate.

This invention concerns the discovery of two distinct PCA3 mRNA sequences. One of these sequences corresponds to a short PCA3 mRNA molecule whereas the other PCA3 RNA molecule is longer as it comprises an additional sequence between exon 3 and exon 4a. The short RNA is associated with prostate cancer whereas the long RNA sequence is associated with a non-malignant state of the prostrate. Based on the differential expression levels of these two PCA3 RNA sequences, protocols for the diagnosis of prostate disease are provided. The invention also relates to therapeutic approaches to prostate cancer.

The present invention relates to systems, methods, and computer program products for the analysis of gene expression data, especially data that have been acquired using microarray technologies. The present invention relates to methods for partitioning a set of genes into clusters, based on the similarity of the genes' rates of messenger RNA synthesis. The present invention also relates to methods for annotating clusters with words or phrases that are extracted from documents associated with genes in the clusters. The present invention also relates to methods for evaluating the quality of clustering based on the extend to which documents associated with genes in a cluster collectively distinguish that cluster from all the other clusters, as well as the extent to which some words and phrases, present in documents associated with genes in the cluster, collectively distinguish that cluster from all the other clusters.

The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized mRNA to a denaturing condition, and purifying the mRNA from the impure preparation from step (a) by tangential flow filtration, wherein the mRNA purified from step (b) is substantially free of prematurely aborted RNA sequences and / or enzyme reagents used in in vitro synthesis.

The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.

The invention relates to an immunoliposome biochip, a preparation method thereof and an application thereof in biological detection. The immunoliposome provided by the invention is distributed on the chip in a micro-array way, and includes molecule probes, such as molecular beacon with fluorescence; and outside of the immunoliposome is loaded with various ligands. The immunoliposome biochip can specifically capture target cells in a cell mixture, and ensure messenger RNA and / or micromolecule RNA in the target cells without destroying biomarkers in the cells. In addition, the immunoliposome biochip provided by the invention can be used for capturing and selecting nanoparticles secreted by virus and cells, such as micro bubbles and exosomes. The immunoliposome biochip has advantages of simple operation, low cost, rapidness, sensitive and high selectivity, and can be used for molecular detection of various diseases, food safety and other fields.