14 results about "Dna cleavage" patented technology

Systems and kits are disclosed herein for genetic engineering (such as for DNA cleavage and gene-editing), which include catalytic nucleic acids and catalytic nucleic acid-assisting reagents. Methods of genetic engineering are also described, in which both catalytic nucleic acid-assisting reagents and catalytic nucleic acids are specific for a target site, thus, providing high-fidelity genetic engineering.

The present disclosure concerns polynucleotides and amino acids of Acidaminococcussp. Casl2a (Cpfl) and methods for their use for genome editing in eukaryotic cells. 20 24 20 14 49 05 M ar 2 02 4 A B S T R A C T 2 0 2 4 2 0 1 4 4 9 0 5 M a r 2 0 2 4

Here, the inventors developed a strategy to modify the chromatin status of HSPCs at the HBG promoters to reactivate HbF expression. In particular, the inventors used from 1 to 4 single guide RNA (sgRNA) molecules spanning from the -220 to the -20 region of the HBG promoters together with CRISPR-based epigenome editors, namely dCas9-CBPcore and Tet1-dCas9. The first editor is capable of inserting histone acetylation while the second editor is performing DNA demethylation. Both epigenetic marks are associated with active transcription and are present in fetal erythroid cells expressing HbF. This strategy allows the ex vivo modification of adult HSCs to recreate a fetal-like epigenetic context leading to HbF production. Moreover, it avoids the genotoxicity associated with classical genome editing tools relying on DNA cleavage. Thus, the present invention relates to a method of increasing the expression of gamma globin in a population of eukaryotic cells through epigenome editing.

Isolation or in vitro assembly of the Cas9-crRNA complex of the Streptococcus thermophilus CRISPR3 / Cas system and use for cleavage of DNA bearing a nucleotide sequence complementary to the crRNA and a proto-spacer adjacent motif. Methods for site-specific modification of a target DNA molecule using an RNA-guided DNA endonuclease comprising at least one RNA sequence and at least one of an RuvC active site motif and an HNH active site motif; for conversion of Cas9 polypeptide into a nickase cleaving one strand of double-stranded DNA by inactivating one of the active sites (RuvC or HNH) in the polypeptide by at least one point mutation; for assembly of active polypeptide-polyribonucleotides complex in vivo or in vitro; and for re-programming a Cas9-crRNA complex specificity in vitro or using a cassette containing a single repeat-spacer-repeat unit.

The present invention relates to modified gRNA molecules, compositions, and methods for site-directed gene editing and genome modification, such as DNA cleavage and gene activation or repression. The modified guide RNA of the present invention has a modified secondary structure (e.g., a long upper stem and a modified hairpin structure) that specifically targets a target DNA sequence having reduced off-target activity.

The present disclosure relates to polynucleotides and amino acids of Acidaminococcus sp. Cas12a (Cpf1) and methods for their use for genome editing in eukaryotic cells.

The application relates to a preparation method and application of an electrochemical sensor for sensitive detection of lead ions based on a metal nanocomposite. 2+ The introduction of the target Pb 2+ triggers the S-DNA cleavage of the DNAzyme to cut off a single-strand DNA, and simultaneously initiates the DNA Walker effect to obtain a large amount of single-strand DNA. The supernatant after magnetic separation is added dropwise on an electrode modified with HP1, after incubation, the single-strand DNA serves as an initiation chain to initiate the CHA signal amplification reaction, so that more and more signal probes are connected to the electrode surface, and the electrical signal in the system is enhanced. The quantitative analysis of lead ions is realized by using the change of the electrical signal corresponding to the change of the methylene blue concentration on the electrode surface before and after the addition of Pb 2+ The prepared electrochemical sensor has high sensitivity, a wide detection range, good selectivity and excellent practical application capability.

The application discloses an ICD inducer Ru(II) complex with a light-driven DNA breaking ability, a preparation method and application thereof, relates to the field of antitumor drugs, and the structural formula of the Ru(II) complex is [Ru(dip)2(L)](PF6)2. The Ru(II) complex can enter a cell nucleus to break DNA and activate immunogenic death mediated by the pyroptosis and cGAS-STING pathways by using photodynamic therapy, so that the antitumor immune effect of the body is improved. In the in-vivo experiment, the immune cells in the tumor microenvironment of the mice in the vaccine group are good, and the antitumor immune effect of the three injections of the vaccine is better. The results of H&E staining show that the vaccine does not cause obvious pathological damage to the main organs of the mice, and the vaccine has good biological safety.

The invention relates to the technical field of tumor immunotherapy, and particularly discloses a screening method of a cancer cell mitochondrial DNA (deoxyribonucleic acid) coding new antigen, mitochondrial DNA with higher purity is obtained by means of microdissection, linear DNA splitting and the like, and nuclear metastasis mitochondrial fragment interference in mitochondrial sequencing is overcome. On the basis, mitochondrial amplicon sequencing is used for identifying the base sequence change caused by mutation from the DNA level, multiple types of tumor vaccines including polypeptide vaccines, RNA vaccines and dendritic vaccines can be prepared according to requirements, and even the tumor vaccines can be used for TCR-T therapy. According to the screening method of the mitochondrial DNA coding new antigen and the application of the mitochondrial DNA coding new antigen in preparation of the vaccine, the feasibility of the mitochondrial coding new antigen and the effectiveness of the mitochondrial coding new antigen as a tumor vaccine target are accurately and comprehensively confirmed, and a transcription translation process and a screening mechanism of DNA-RNA-polypeptide are systematically constructed; and more diversified, personalized, safe and effective treatment strategies can be selected and designed for the patient.

This invention provides a method for detecting microRNA using thermophilic TtAgo protein based on cofactor activation of its mesophilic DNA cleavage activity. This invention expands the applicable temperature range of TtAgo by using a cofactor that extends the function of the extremely thermophilic TtAgo protein and activates its mesophilic DNA cleavage activity, thereby establishing a one-step closed-tube isothermal multiplex analysis method for microRNA based on TtAgo that is not limited by high temperatures.

Described herein are CAS12A mutants from Lachnospiraceae bacterium and methods for use thereof. These mutants have enhanced DNA cleavage activities at non-canonical TTTT protospacer adjacent motifs (PAM) compared to the wild-type enzyme.

The present invention relates to an optimized technology for regulating target-specific gene expression based on an ultra-miniaturized OMEGA system, and a gene expression regulatory system that can enhance gene regulatory efficiency using TnpB is constructed. In the present invention, mutated TnpB with an inactivated DNA cleavage function, or engineered reRNA was developed, establishing the optimal conditions that can yield gene expression efficiency using original TnpB without any introduced mutations, and the vector for regulating gene expression of the present invention is small enough to be loaded in AAVs. Thus, it can be effectively used in a gene expression regulation technology.

The present disclosure relates to DNA-PK inhibitors of formula (I), (I), or a pharmaceutically acceptable salt, stereoisomer, solvate, prodrug, or tautomer thereof, methods of making the foregoing and compositions thereof, and methods of use of compounds of formula (I) in combination with DNA cleavage agents.

The present invention relates to modified gRNA molecules, compositions and methods for site-specific gene editing and genome modification, such as DNA cleavage and gene activation or inhibition. In some embodiments, the modified guide RNAs have modified secondary structures (e.g., long upper stems and modified hairpin structures) that specifically target a target DNA sequence with reduced off-target activity.