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32 results about "Genome engineering" patented technology
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Genome engineering refers to the strategies and techniques developed in recent years for the targeted, specific modification of the genetic information – or genome – of living organisms. It represents a very active field of research because of the wide range of possible applications, particularly in the areas of human health - the correction of a gene carrying a harmful mutation, the production of therapeutic proteins, the elimination of persistent viral sequences - agricultural biotechnology - the development of new generations of genetically modified plants - and for the development of research tools - for example, to explore the function of a gene. Early technologies developed to insert a gene into a living cell, such as transgenesis, are limited by the random nature of the insertion of the new sequence into the genome. The new gene is positioned blindly, and may inactivate or disturb the functioning of other genes or even cause severe unwanted effects; it may trigger a process of cancerization, for example. Furthermore, these technologies offer no degree of reproducibility, as there is no guarantee that the new sequence will be inserted at the same place in two different cells.
Methods for enhancing genome engineering efficiency
This document relates to methods and materials for genome engineering in eukaryotic cells, and particularly to methods for increasing genome engineering (i.e. transformation or genome editing) efficiency via delivery of one or more RKD2 and RKD4 genes, with genome engineering components.
Owner:UNIVERSITY OF WARWICK
High fidelity nucleotide polymerase chimeric prime editor systems
The present invention relates to the field of genomic engineering. In particular, a chimeric prime editing (cPE) system is disclosed comprising elements including, but not limited to a Cas9 nickase (nCas9) / high fidelity nucleotide polymerase (HFNTPol) RNA, one or more single guide RNAs (sgRNAs), and a chimeric prime editor template oligonucleotide (cpetODN) comprising a deoxyribonucleic acid nucleotide polymerase template (NPT) and a primer binding site. For example, the sgRNA and the cpetODN are ligated into a single oligonucleotide. Alternatively, the sgRNA and the cpetODN are free and independent molecules (e.g., modular). This cPE system results in precise and efficient genome editing in cells and in adult mouse liver which is advantageous over conventional sgRNA prime editor fusion constructs. This flexible and modular system is an improvement in the art to obtain precise genome editing.
Owner:UNIV OF MASSACHUSETTS
Vector comprising gene associated with DNA repair deficiency and uses thereof
The present disclosure relates, in general, to expression vectors comprising genes associated with DNA repair deficiencies, such as Fanconi anemia (FA), for use in gene therapy to treat DNA repair deficiency disorders. The expression vectors can be introduced directly into the patients or used ex vivo in a method for genome engineering a hematopoietic stem and progenitor cell (HSPC) to express a gene of interest and reintroduced into a patient.
Owner:REGENTS OF THE UNIVERSITY OF MINNESOTA
Engineering AAV
The present disclosure provides methods and compositions to develop AAV capsids with a desired characteristic compared to a natural AAV serotype. These capsids are useful, for example, for the delivery of genome engineering molecules and gene therapy molecules for the treatment of a subject in need thereof.
Owner:SANGAMO THERAPEUTICS INC
Methods and compositions for generating polynucleotides
PCT designated stageWO2026006560A3HydrolasesVector-based foreign material introductionPolynucleotideGenome
Recombinant nucleic acids, compositions and methods for producing polynucleotides, such as donor sequences, as well as their use in a variety of applications including genome engineering.
Owner:LIFE TECHNOLOGIES CORP
Ipsc-derived t cells for solid tumor therapy
PendingCN122249220AAntibody mimetics/scaffoldsInorganic active ingredientsDirected differentiationEffector cell
This invention provides methods and compositions for cancer immunotherapy. In various embodiments, the composition comprises functionally enhanced derived effector cells obtained by directed differentiation of genome-engineered iPSCs. In various embodiments, the derived cells provided herein have stable and functional genome editing that delivers improved or enhanced therapeutic effects. Therapeutic compositions and their uses are also provided, comprising these functionally enhanced derived effector cells alone or in combination therapies.
Owner:FATE THERAPEUTICS INC
Method for generating regulatory T cells (TREGs) using genome engineering
Methods, polynucleotides, and compositions for generating engineered Treg cells are provided. The methods, polynucleotides, and compositions enable the reprogramming of hematopoietic cells into Treg cells by constitutive or controlled expression of FOXP3 in engineered cells, so that engineered Treg cells can suppress the activation and proliferation of responder T cells.
Owner:LUNG BIOTECH PBC
Lipid nanoparticles compositions with ribonucleoproteins
Provided herein are lipid nanoparticles comprising a ribonucleoprotein complex (RNP) comprising a Type II Cas nuclease polypeptide (e.g., a Cas9 polypeptide) and a guide RNA (gRNA), e.g., a single guide RNA (sgRNA). Compositions and formulations comprising such lipid nanoparticles are also provided. Such lipid nanoparticles can be used for delivery of a RNP to a cell, for example, a cell that is present in a subject in vivo. Methods of producing such lipid nanoparticles, and methods of use for genome engineering, are also provided.
Owner:INTELLIA THERAPEUTICS INC
CD3 reconstitution in engineered iPSC and immune effector cells
Provided are methods and compositions for obtaining functionally enhanced derivative effector cells obtained from directed differentiation of genomically engineered iPSCs. The iPSC-derived cells provided herein have stable and functional genome editing that delivers improved or enhanced therapeutic effects. Also provided are therapeutic compositions and the used thereof comprising the functionally enhanced derivative effector cells alone, or with antibodies or checkpoint inhibitors in combination therapies.
Owner:FATE THERAPEUTICS INC
Compositions and methods of nucleic acid targeting nucleic acid
The present disclosure provides compositions and methods of use of nucleic acids and complexes thereof targeted to nucleic acids. Genomic engineering can refer to the alteration of a genome by deletion, insertion, mutation, or replacement of a particular nucleic acid sequence. The alteration can be gene or position specific. Genomic engineering can utilize nucleases to cleave nucleic acids, thereby generating a site for alteration. Engineering of non-genomic nucleic acids is also contemplated.
Owner:CARIBOU BIOSCIENCES INC
System for preparing target DNA, expression vector composition and application thereof
PendingCN121950876AAchieve autonomous and controllable generationEnables controlled modificationBacteriaVirus peptidesBase JReverse transcriptase
The invention discloses a system for preparing target DNA, an expression vector composition and application thereof, and relates to the technical field of biology. The system for gene editing comprises: a bacteriophage activating protein; and a DRT2 system or a reorganized DRT2 system; the bacteriophage activating protein comprises at least one of single-stranded DNA annealing protein, single-stranded DNA binding protein and ribonucleotide reductase; the DRT2 system comprises a gene for coding reverse transcriptase and ncRNA; the recombinant DRT2 system is obtained by the following method: replacing the 45th to 104th basic groups of ncRNA in the DRT2 system with a target gene. According to the invention, the DRT2 system or the recombinant DRT2 system is activated by utilizing the phage activating protein, and then the target double-stranded DNA is generated through reverse transcription without depending on an exogenous infection signal, so that the system has relatively strong applicability in gene editing and genome engineering.
Owner:SOUTHERN UNIVERSITY OF SCIENCE AND TECHNOLOGY
Effector cells having enhanced target cell recognition
PCT designated stageWO2026102183A1Polypeptide with localisation/targeting motifImmunoglobulin superfamilyDirected differentiationEffector cell
Provided are methods and compositions for obtaining functionally enhanced immune cells, and derivative effector cells obtained from directed differentiation of genomically engineered iPSCs. Also provided are derivative cells having stable and functional genome editing that delivers improved or enhanced therapeutic effects. Further provided are therapeutic compositions and the use thereof comprising the functionally enhanced derivative effector cells alone, or with antibodies or checkpoint inhibitors in combination therapies.
Owner:FATE THERAPEUTICS INC
Genomic safe harbor region of long clam for stable integration of exogenous genes and screening method
PendingCN122303247AOstrea gigasChromosomal region
This invention belongs to the field of molecular genetics and shellfish genome engineering, and particularly relates to a safe harbor region of the oyster genome for stable integration of exogenous genes and a screening method. The safe harbor region is a region for stable integration and expression of exogenous genes, located in different chromosomal regions of the oyster genome, in a non-open chromatin state, and meeting the following conditions: (1) the region is located on chromosomes and scaffolds, and has no coding genes / lncRNAs; (2) the region has no ATAC-seq signal; (3) the region length is 5.25–14.84 kb; (4) the average methylation level of the region is 0–0.161765; (5) the PAM (NGG) density of the region is 38.68–81.35 PAM / kb. This invention provides a reproducible and scalable general technical platform for the stable expression of oyster gene knock-in fragments and the study of their gene function.
Owner:OCEAN UNIV OF CHINA
Construction method and application of a sypl1 gene knockout colorectal cancer mouse model
The application relates to the technical field of animal model construction, and particularly discloses a Sypl1 gene knockout colorectal cancer mouse model construction method and application, which comprises the following steps: synthesizing a specific target site gRNA of a Sypl1 gene, mixing Cas9 protein and the target site gRNA to obtain an injection compound, microinjecting the injection compound into mouse zygotes, and transplanting the surviving zygotes into the oviducts of pseudopregnant female mice to obtain F0 generation mice, and the mice born after 20 days are Sypl1 gene knockout mouse animal models; and a chemical induction modeling method is used to construct a colorectal cancer model in wild type mice and gene knockout mice. The Sypl1 gene knockout mouse is constructed by using a CRISPR / Cas mediated genome engineering technology Sypl1 combined with the most widely used colorectal cancer chemical inducer azoxymethane / dextran sodium sulfate, a new colorectal cancer mouse model is constructed, compared with a traditional chemical induction model, the tumor formation rate can be significantly improved, and the tumor formation time can be shortened, and a more efficient animal model is provided for colorectal cancer research.
Owner:THE THIRD PEOPLES HOSPITAL OF CHENGDU
Construction method and application of Sypl1 gene knockout colorectal cancer mouse model
The invention relates to the technical field of animal model construction, and particularly discloses a construction method and application of a Sypl1 gene knockout colorectal cancer mouse model, and the construction method comprises the following steps: synthesizing a specific target site gRNA of a Sypl1 gene, uniformly mixing Cas9 protein and the target site gRNA to obtain an injection compound, microinjecting the injection compound into a mouse fertilized egg to obtain a Sypl1 gene knockout colorectal cancer mouse model. And transplanting the survival fertilized ova into the salpingtube of a pseudopregnant female mouse for 20 days to obtain a mouse which is an F0-generation mouse, obtaining a Sypl1 gene knockout mouse animal model, and constructing a colorectal cancer model in a wild type mouse and a gene knockout mouse by adopting a chemical induction modeling method. A Sypl1 gene knockout mouse is constructed through a CRISPR / Cas mediated genome engineering technology, a novel colorectal cancer mouse model is constructed in combination with a colorectal cancer chemical inducer azomethane / dextran sodium sulfate which is most widely applied at present, and compared with a traditional chemical induction model, the novel colorectal cancer mouse model has the advantages that the tumor formation rate can be remarkably increased, the tumor formation time can be shortened, and the tumor formation cost can be reduced. And a more efficient animal model is provided for colorectal cancer research.
Owner:THE THIRD PEOPLES HOSPITAL OF CHENGDU
Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
ActiveUS12649913B2Fusion with DNA-binding domainAntibody mimetics/scaffoldsGenomic engineeringPrevotella
Engineered CRISPR from Prevotella and Francisella 1 (Cpf1) nucleases with altered and improved target specificity and their use in genomic engineering, epigenomic engineering, genome targeting, genome editing, and in vitro diagnostics.
Owner:THE GENERAL HOSPITAL CORP
Engineered proteins
This invention provides a designed protein that can be used as a genome engineering tool. The protein of this invention has, in the amino acid sequence represented by SEQ ID NO: 1, a substitution at amino acid position 188 to histidine, and further has one substitution selected from the following: a substitution at amino acid position 2 to tyrosine, a substitution at amino acid position 70 to tyrosine, a substitution at amino acid position 80 to arginine, a substitution at amino acid position 105 to threonine, a substitution at amino acid position 123 to histidine, a substitution at amino acid position 195 to lysine, a substitution at amino acid position 208 to arginine, a substitution at amino acid position 232 to alanine, a substitution at amino acid position 246 to methionine, a substitution at amino acid position 316 to methionine, or a substitution at amino acid position 337 to isoleucine.
Owner:THE UNIV OF TOKYO +3
Genome editing using programmable unwinding-annealing helicase and related single-strand annealing proteins
PCT designated stageWO2026050362A1Organic active ingredientsPeptide/protein ingredientsSingle strandHelicase
Provided herein are compositions, systems, and methods for genome engineering using programmable unwinding-annealing helicase and related single-strand DNA annealing proteins (SSAPs), for example, from eukaryotic cells.
Owner:THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV
Bacterial multi-target non-specific genome engineering method and application thereof
The invention discloses a bacterium multi-target non-specific genome engineering method and application thereof, and relates to the field of gene engineering. According to the invention, the MTI system is successfully expanded from eukaryotic cells to a prokaryotic bacterium system for the first time, the dependence on a long-segment specific attachment site (attB) is thoroughly eliminated in mechanism, and any pre-modified specific sequence does not need to be carried out on a host genome, so that real host-independent editing is realized. On the basis, a set of genome engineering platform which is simple and convenient to operate and universal is constructed, the platform supports stable integration of multi-copy and large-fragment DNA and is successfully applied to various bacteria including gram-positive bacteria and gram-negative bacteria, and the operable host range of genome engineering is greatly expanded.
Owner:SHANGHAI JIAOTONG UNIV
Genetic transformation and genome engineering in legume species
Methods and materials for generating leguminous plant tissue having one or more genetic modifications of interest are provided herein. For example, methods and materials that use developmental regulators (DRs) to improve the transformation process and efficiency in legumes are provided herein.
Owner:REGENTS OF THE UNIVERSITY OF MINNESOTA
Peptide conjugates and use thereof to promote CAS nuclease immune tolerance in genome engineering gene therapy
In the field of, the use of polypeptide conjugates, compositions and methods for promoting immune tolerance to the CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR associated protein) system, for genome engineering gene therapy. The polypeptide conjugates includes a first component, which is an antigen-presenting cell antibody, and a second component, which is a Cas protein.
Owner:ASFALIA BIOLOGICS
Compositions and methods for crispr / cas9 based reactivation of human angelman syndrome
PCT designated stageWO2026006542A3Organic active ingredientsSpecial deliveryGenomic mutationGenetics
Systems and methods for highly-effective CRISPR-Cas based genomic editing within human chromosome 15q11-q13 have been developed as therapeutic interventions for Angelman Syndrome (AS). Selective single guide RNA molecules (sgRNAs) that impart enhanced CRISPR-Cas editing of genomic mutations associated with Angelman Syndrome in human cells are described. The engineered sgRNAs induce activity of non-pathogenic, paternal UBE3A alleles to reduce, reverse and / or prevent the causative neurodevelopmental defects of AS in a subject in need thereof. Compositions and methods of engineered crRNAs, sgRNAs thereof and ribonucleoprotein (RNP) complexes thereof are provided for enhanced genomic engineering with increased on-off target specificity and on-target editing efficacy for treatment of AS.
Owner:YALE UNIVERSITY
Variants of CRISPR from Prevotella and Francisella 1 (Cpf1)
ActiveUS12590299B2Fusion with DNA-binding domainAntibody mimetics/scaffoldsGenomic engineeringGenus Francisella
Engineered CRISPR from Prevotella and Francisella 1 (Cpf1) nucleases with altered and improved target specificity and their use in genomic engineering, epigenomic engineering, genome targeting, genome editing, and in vitro diagnostics.
Owner:THE GENERAL HOSPITAL CORP
Recombinase combination suitable for bacteroides, application and homologous recombination method
PendingCN122012630AMicroorganism based processesStable introduction of DNABiotechnologyEscherichia coli
The invention discloses a recombinase combination suitable for bacteroides, application and a homologous recombination method, and belongs to the technical field of microbial genetic engineering. The invention discloses a method for constructing and screening a combination of a single-chain annealing protein and a single-chain binding protein for the first time. A single-chain annealing protein and a single-chain binding protein are jointly constructed in an escherichia coli-bacteroides fragilis shuttle expression vector, and co-expression of the escherichia coli-bacteroides fragilis shuttle expression vector in bacteroides fragilis is achieved, so that the homologous recombination capacity of bacteroides fragilis is remarkably improved. According to the method disclosed by the invention, the homologous recombination efficiency of genome editing of bacteroides fragilis is greatly improved, the absolute editing efficiency of the genome is improved to 0.052% from the original level which is almost undetectable, and the research and application progress of genome engineering of the strain is remarkably promoted.
Owner:SHANDONG UNIV +1
Methods and compositions for promoting non-natural amino acid-containing protein production
Disclosed herein are methods and compositions for generation of cell lines to promote unnatural amino acid-containing protein production using genome engineering technology.
Owner:AMBRX INC
Lipid nanoparticles compositions with ribonucleoproteins
PCT designated stageWO2026117712A2Powder deliveryMicroencapsulation basedRibonucleoprotein complexNanoparticle
Provided herein are lipid nanoparticles comprising a ribonucleoprotein complex (RNP) comprising a Type II Cas nuclease polypeptide (e.g., a Cas9 polypeptide) and a guide RNA (gRNA), e.g., a single guide RNA (sgRNA). Compositions and formulations comprising such lipid nanoparticles are also provided. Such lipid nanoparticles can be used for delivery of a RNP to a cell, for example, a cell that is present in a subject in vivo. Methods of producing such lipid nanoparticles, and methods of use for genome engineering, are also provided.
Owner:INTELLIA THERAPEUTICS INC
Genome Engineering
Methods are provided for altering target DNA in a cell genetically modified to express a Cas 9 enzyme that forms a co-localization complex with a guide RNA complementary to the target DNA and that cleaves the target DNA in a site specific manner. Methods include introducing into the cell a first foreign nucleic acid encoding a donor nucleic acid sequence, introducing into the cell from media surrounding the cell the guide RNA complementary to the target DNA and which guides the Cas 9 enzyme to the target DNA, wherein the RNA and the enzyme are members of a co-localization complex for the target DNA, wherein the donor nucleic acid sequence is expressed, wherein the guide RNA and the Cas 9 enzyme co-localize to the target DNA, the Cas 9 enzyme cleaves the target DNA and the donor nucleic acid is inserted into the target DNA to produce altered DNA in the cell.
Owner:PRESIDENT & FELLOWS OF HARVARD COLLEGE
IPSC-derived effector cells for treatment of autoimmune diseases
PendingCN121752276AOrganic active ingredientsImmunoglobulin superfamilyAutoimmune conditionAutoimmune disease
Methods and compositions for immunotherapy are provided. In various embodiments, the compositions comprise functionally enhanced derived effector cells obtained by directed differentiation of genome engineered iPSCs. In various embodiments, the derived cells provided herein have stable and functional genome editing that delivers improved or enhanced therapeutic effects. Also provided are therapeutic compositions comprising derived effector cells, alone or enhanced in these functions in combination therapy, and uses thereof.
Owner:FATE THERAPEUTICS INC
Solid tumor targeting scaffolds to promote effector cell differentiation and function
PendingCN121794368AImmunoglobulin superfamilyGenetically modified cellsMedicineDirected differentiation
Methods and compositions are provided for obtaining functionally enhanced immune cells as well as derived effector cells obtained by directed differentiation of genome engineered iPSCs. Also provided are stable and functional genome-edited derived cells that deliver improved or enhanced therapeutic effects. Further provided are therapeutic compositions and uses thereof, the therapeutic compositions comprising the functionally enhanced derived effector cells alone, or comprising the functionally enhanced derived effector cells in combination therapy with an antibody or checkpoint inhibitor.
Owner:FATE THERAPEUTICS INC