Patent classifications
C12N2310/128
A UNIVERSAL PLATFORM TO ENHANCE CRISPR-BASED GENE EDITING FOR IN VIVO THERAPIES
Aspects of the disclosure relate to methods and synthetic regulatory systems for more efficient nuclease-mediated homology-directed repair (HDR). In particular, provided herein are methods for more efficient in vivo and in vitro HDR-based gene editing where the methods comprise introducing into a cell a synthetic regulatory system comprising Cas nuclease, guide RNAs (gRNAs) having various lengths and configured to target distinct nucleotide sequences for simultaneous transcriptional repression (or activation) and genome editing via double stranded break and use of a donor nucleic acid molecule as a template for repair.
METHOD OF TREATING TYPE II DIABETES AND OBESITY AND METHOD OF SCREENING A MEDICAMENT FOR THE SAME
The present invention provides a use of an agent for preparing a medicament for inhibiting microRNA-708. The medicament is used for at least one of the following: reducing intracellular triglyceride content, inhibiting differentiation of fat cells, resisting obesity, promoting insulin sensitivity, increasing respiratory metabolic rates, increasing energy consumption, increasing the number of mitochondria, up-regulating oxidative phosphorylation or heat-producing genes, relieving insulin resistance, resisting fatty liver and treating or preventing Type 2 diabetes.
Simultaneous multiplex genome editing in yeast
The present disclosure provides compositions of matter, methods and instruments for editing nucleic acids in live yeast cells.
SIMULTANEOUS MULTIPLEX GENOME EDITING IN YEAST
The present disclosure provides compositions of matter, methods and instruments for editing nucleic acids in live yeast cells.
A UNIVERSAL PLATFORM TO ENHANCE CRISPR-BASED GENE EDITING FOR IN VIVO THERAPIES
Aspects of the disclosure relate to methods and synthetic regulatory systems for more efficient nuclease-mediated homology-directed repair (HDR). In particular, provided herein are methods for more efficient in vivo and in vitro HDR-based gene editing where the methods comprise introducing into a cell a synthetic regulatory system comprising Cas nuclease, guide RNAs (gRNAs) having various lengths and configured to target distinct nucleotide sequences for simultaneous transcriptional repression (or activation) and genome editing via double stranded break and use of a donor nucleic acid molecule as a template for repair.
METHOD FOR SELECTIVE TRANSLATION OF DESIRED PROTEINS IN THE RESENCE OF A SPECIFIED NUCLEIC ACID LIGAND
There is a method for selective translation of a desired protein. The method has the steps of (a) providing two half cores of a deoxyribozyme, wherein each half core includes a pendant assembly arm of a strand of nucleic acids extending therefrom and a separate, pendant binding arm extending therefrom of a strand of nucleic acids; (b) binding a nucleic acid ligand to each of the two assembly arms to form an intermediate; (c) binding the intermediate to (i) a first substrate of ribonucleic acid sequences capped at one end, (ii) a second substrate of a strand of ribonucleic acids having a 5 triphosphate region at one end and a region of polyadenylated nucleotides at the other end and wherein the second substrate codes for the desired protein, (iii) join the two half cores to form a core in order to form a ligated product; and (d) allowing the translation for the desired protein to proceed from the ligated product. There is another method for selective translation of a desired protein. There is also a modified nucleic acid enzyme.
RNA MOLECULES, METHODS OF PRODUCING CIRCULAR RNA, AND TREATMENT METHODS
The present invention relates to a RNA molecule comprising a first ribozyme, a first ligation sequence, an effector molecule, a second ligation sequence, and a second ribozyme. Methods of producing circular RNA molecules and treatment methods are also disclosed.
Photoreversible Acylation Reagents
Reagents and methods to cloak and uncloak RNA polymers and applications thereof are provided. Photocloaking molecules are used to label RNA polymers. Radiant energy is used to remove photoreleaseable protecting adducts and revert a RNA polymer to its native form.
Cancer specific-splicing ribozyme and use thereof
The present invention relates to a recombination vector, a transformation cell into which the recombinant vector is introduced, a ribozyme expressed from the recombination vector, a prophylactic or therapeutic composition for liver cancer comprising the recombination vector and the ribozyme, and a therapeutic method for liver cancer using the composition, said recombination vector comprising: a tissue-specific promoter; and a ribozyme-target gene expression cassette comprising a trans-splicing ribozyme targeting a cancer-specific gene and a target gene connected to the 3 exon of the ribozyme, wherein a splicing donor/splicing acceptor sequence (SD/SA sequence) is connected to the 5 end of the ribozyme-target gene expression cassette, woodchuck hepatitis virus posttranscriptional regulatory element (WPRE) is connected to the 3 end of the ribozyme-target gene expression cassette, and a nucleic acid sequence recognizing a micro RNA-122a (microRNA-122a, miR-122a) is further connected to the 3 end of the WPRE.
ADAPTIVE CARE AREAS FOR DIE-DIE INSPECTION
The present disclosure describes methods, systems, and articles of manufacture for performing a defect inspection of a die image using adaptive care areas (ACAs). The use of ACAs solve the problem of handling rotations of components that require rotating care areas; handling the situation where each care area requires its own rotation, translation, or affine transformation; and the situation of decoupling intensity differences caused by defects or process variation from intensity differences caused by size variations.