Patent classifications
C12N15/10
Genome fractioning
Disclosed herein are compositions and methods related to the elimination of molecules of a selected sequence from a nucleic acid sample or from an sequence dataset resulting from the sequencing of a sample, for example to exclude such molecules from downstream analysis or sequencing, or to exclude such sequences from a downstream data set.
TARGETING BCL11A ENHANCER FUNCTIONAL REGIONS FOR FETAL HEMOGLOBIN REINDUCTION
Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.
Compositions and Methods for Treating Huntington's Disease and Related Disorders
Compositions and methods are provided for the inhibition, treatment and/or prevention of Huntington's disease and related disorders.
GENE EDITING METHODS AND COMPOSITIONS FOR ELIMINATING RISK OF JC VIRUS ACTIVATION AND PML (PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY) DURING IMMUNOSUPPRESSIVE THERAPY
A method of eliminating the risk of JCV activation in a subject undergoing immunosuppressive therapy, by administering an effective amount of a gene editing composition directed toward at least one target sequence in the JCV genome, cleaving the target sequence in the JCV genome, disrupting the JCV genome, eliminating the JCV infection, eliminating the risk of JCV activation, and treating the subject with an immunosuppressive therapy. A pharmaceutical composition including at least one isolated nucleic acid sequence encoding a CRISPR-associated endonuclease and at least one gRNA having a spacer sequence complementary to a target sequence in a JCV DNA, the isolated nucleic acid sequences being included in at least one expression vector. Pharmaceutical compositions including at least one isolated nucleic acid sequence encoding at least one TALEN, at least one ZFN, and gene editing composition of C2c1, C2c3, TevCas9, Archaea Cas9, CasY.1-CasY.6, CasX, or argonaute protein, which target at least one nucleotide sequence of the JCV genome.
METHODS AND MEANS FOR GENETIC ALTERATION OF GENOMES UTILIZING DESIGNER DNA RECOMBINING ENZYMES
The invention provides methods and means for specifically altering the DNA sequence in a genome, in particular for genome editing by deleting or replacing a sequence of interest. Advantageously, the invention uses two non-identical sequences naturally occurring in a genome as target sites two which DNA-recombining enzymes are generated. The invention is in particular useful for medicine, in particular to repair a mutation in a genome or to delete predefined genetic material from cells or tissue and to cure diseases. An advantage of the invention is that it allows precise site directed altering of DNA without engaging host DNA repair pathways and thereby works without inducing random insertions and deletions (in-dels).
DEVICE FOR EXTRACTING A NUCLEIC ACID FROM A SAMPLE LIQUID
A device for extracting a nucleic acid from a sample liquid includes a heating element configured to be connected to an extraction nucleic acid. The extraction nucleic acid is at least partly complementary to the nucleic acid to be extracted from the sample liquid. The heating element is heatable to a temperature that is equal to or higher than a denaturing temperature of the nucleic acid bound to the extraction nucleic acid.
OLIGONUCLEOTIDE ENCODED CHEMICAL LIBRARIES
This application provides a bead with a covalently attached chemical compound and a covalently attached DNA barcode and methods for using such beads. The bead has many substantially identical copies of the chemical compound and many substantially identical copies of the DNA barcode. The compound consists of one or more chemical monomers, where the DNA barcode takes the form of barcode modules, where each module corresponds to and allows identification of a corresponding chemical monomer. The nucleic acid barcode can have a concatenated structure or an orthogonal structure. Provided are method for sequencing the bead-bound nucleic acid barcode, for cleaving the compound from the bead, and for assessing biological activity of the released compound.
KITS FOR ANALYSIS USING NUCLEIC ACID ENCODING AND/OR LABEL
Kits and methods of using the kits for analyzing macromolecules, including peptides, polypeptides, and proteins, employing nucleic acid encoding are disclosed. The sample analysis kits employ nucleic acid encoding and/or nucleic acid recording of a molecular interaction and/or reaction, such as recognition events (e.g., between an antigen and an antibody, between a modified terminal amino acid residue, or between a small molecule or peptide therapeutic and a target, etc.). Additional barcoding reagents, such as those for cycle-specific barcoding (e.g., “clocking”), compartment barcoding, combinatorial barcoding, spatial barcoding, or any combination thereof, may be included in the kits. The sample may comprise macromolecules, including peptides, polypeptides, and proteins, and the recording may generate molecular interaction and/or reaction information, and/or polypeptide sequence information. The kits may be used in high-throughput, multiplexed, and/or automated analysis, and are suitable for analysis of a proteome or subset thereof.
A HTP PLATFORM FOR THE GENETIC ENGINEERING OF CHINESE HAMSTER OVARY CELLS
Presented herein is a high-throughput (HTP) genomic engineering platform for improving the production of therapeutic proteins in Chinese hamster ovary (CHO) cells. The disclosed HTP genomic engineering platform is computationally driven and integrates molecular biology, automation, and advanced machine learning protocols. The platform utilizes a unique suite of HTP genetic engineering tools to explore the genomic landscape associated with therapeutic protein production pathways, in order to unravel the biological drivers and disentangle the uncharacterized genetic architecture responsible for optimizing therapeutic protein production in CHO cells.
PROCESS FOR EXTRACTING DOUBLE-STRANDED DNA
The present invention relates to a process for extracting double-stranded DNA, using ionic liquids of formula (I). It also relates to kits comprising such ionic liquids to implement such a process.