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73 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.
Compositions and methods for crispr / CAS9 based reactivation of human angelman syndrome
PCT designated stageWO2026006542A2Organic 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
Efficient homologous recombination method suitable for bifidobacteria and application of efficient homologous recombination method
PendingCN120192981AFusion with DNA-binding domainStable introduction of DNABiotechnologyEscherichia coli
The invention belongs to the field of microbial genetic engineering, and particularly relates to an efficient homologous recombination method suitable for bifidobacteria and application of the efficient homologous recombination method. Specifically, the invention discloses a method for constructing and screening the combination of the single-chain annealing protein and the single-chain binding protein for the first time. According to the method, a single-chain annealing protein and a single-chain binding protein are jointly constructed in an escherichia coli-bifidobacterium shuttle expression vector, and co-expression of the single-chain annealing protein and the single-chain binding protein in bifidobacterium longum is realized, so that the homologous recombination capability of the bifidobacterium longum is remarkably improved. According to the method disclosed by the invention, the homologous recombination efficiency of genome editing of the bifidobacterium longum is greatly improved, the absolute genome editing efficiency of the bifidobacterium longum is improved to 0.00009792% from the original level which is almost undetectable, and the research and application progress of genome engineering of the strain is remarkably promoted, so that the method has a good practical application value.
Owner:SHANDONG UNIV
Systems and methods for increased production of recombinant biopolymers via genome engineering and downregulation of basal expression
Recombinant E. coli strains and synthetic protein sequence designs are leveraged for production of disordered polypeptides such as spidroins and elastin-like peptides (ELPs). These disordered polypeptides, the high-titer production of which has proven difficult, include repeating structural motifs from a small selection of amino acid residues, resulting in lack of well-defined tertiary and quaternary structure. The recombinant E. coli include expression vectors with genes encoding for the disordered polypeptide product. Expression of these genes is controlled by a promoter that downregulates and substantially inhibits basal expression in the recombinant bacteria. Further, the recombinant bacteria include mutations to one or more stress-response genes from wild-type E. coli, such as yggw, yedv, yedw, yedy, spec, speb, uspc, hcha, loip, mltc, envz, ompr, yhgf, or hupb. The recombinant E. coli enable production of high titers of disordered protein product while minimizing the toxic effects thereof on the host.
Owner:RENESSELAER POLYTECHNIC INST
Gene editing using homology-independent universal genome engineering technology
Disclosed herein are the genetic constructs for a Homology-Independent Universal Genome Editing (HiUGE) system and methods of using said HiUGE system for genome editing. The invention relates to compositions comprising gRNA polynucleotides, insert polynucleotides, and a CRISPR-based nuclease or polynucleotide encoding a CRISPR-based nuclease.
Owner:DUKE UNIV
A CRISPR-Cas9 plasmid and its construction method and application
The present invention relates to a CRISPR-Cas9 plasmid and its construction method and application. The nucleotide sequence of the CRISPR-Cas9 plasmid is shown in SEQ ID No. 6. A CRISPR-Cas9 plasmid for Bifidobacterium was constructed using CRISPR-Cas9 technology. By transforming Bifidobacterium with this plasmid, a gene knockout strain can be obtained, which is conducive to more convenient and efficient application in Bifidobacterium genome engineering.
Owner:UNIV OF SHANGHAI FOR SCI & TECH
Construction method and application of animal model for research on HPV pathogenic mechanism
The invention discloses a construction method and application of an animal model for HPV (human papillomavirus) pathogenic mechanism research, and belongs to the technical field of animal models. According to the construction method of the HPV fixed-point integration animal model, HPV16 gene knock-in is carried out on the Col1a1 gene locus of a C57BL / 6J mouse through CRISPR / Cas mediated genome engineering. The method comprises the following steps: (1) designing specific gRNA; (2) constructing a knock-in vector Donor vector; and (3) uniformly mixing Cas9 protein, gRNA and a targeting vector Donor vector to obtain an injection compound, performing micro-injection on the injection compound into a mouse fertilized egg, and sending the injection compound back to a pregnant mouse fallopian tube to cultivate offspring so as to generate a gene knock-in mouse. The animal model prepared by the method can be used as a contrast of a cervical cancer precancerous lesion animal model for scientific research.
Owner:TONGJI HOSPITAL ATTACHED TO TONGJI MEDICAL COLLEGE HUAZHONG SCI TECH
Methods and compositions for improving precision of endonuclease-mediated genome editing
PCT designated stage expiredWO2025117745A1Cell receptors/surface-antigens/surface-determinantsStable introduction of DNACell therapyEndonuclease
Disclosed are methods and compositions for reducing concatemer formation and formation of other artifacts during insertion of donor nucleic acid into a genome. Applications to genome editing and genome-engineered call therapy are also disclosed.
Owner:CARIBOU BIOSCIENCES INC
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
Enhanced immune effector cells and use thereof
Provided are methods and compositions for obtaining functionally enhanced derivative effector cells obtained from directed differentiation of genomically engineered iPSCs. The derivative 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
PEPTIDE CONJUGATES AND THEIR USE TO PROMOTE IMMUNOTOLERANCY TO CAS NUCLEASES IN GENOME ENGINEERING GENE THERAPY
PEPTIDE CONJUGATES AND THEIR USE FOR PROMOTING IMMUNOTOLERANCY TO CAS NUCLEASES IN GENOME ENGINEERING GENE THERAPY The present invention relates generally to the field of medicine. More particularly, it relates to polypeptide conjugates, compositions, and methods for promoting immunotolerance to the CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR associated protein) system, for gene therapy by genome engineering. (No figure)
Owner:ASFALIA BIOLOGICS
Cells having solid tumor targeting backbone and use thereof
Provided are methods and compositions for obtaining functionally enhanced 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
Cellular reprogramming using temporal and transient plasmid vector expression system
Provided are methods and compositions for inducing the reprogramming of a non-pluripotent to an iPSC having desirable properties using a vector system providing transient and temporal expression of transgenes that are short-lived. Also provided are reprogramming cells and iPSC populations or clonal cell lines using the provided reprogramming methods and compositions. Further provided are genome-engineered iPSCs and derived cells redifferentiated therefrom to comprise targeted editing involving insertions and deletions in one or more selected genomic loci.
Owner:FATE 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
Methods of assessing engineered retron activity, and uses thereof
PendingUS20250223586A1Microbiological testing/measurementVector-based foreign material introductionRetroposonEngineering
Methods and compositions are described herein that are useful for analyzing and identifying retron systems with improved replication fidelity and efficiency. The methods and compositions described herein facilitate the engineering, quantification, and identification of retron elements for precise genome engineering.
Owner:THE J DAVID GLADSTONE INSTITUTES
Engineering native e. coli for tumor imaging under hypoxia conditions
PCT designated stageWO2025217633A1BacteriaBacteria material medical ingredientsGastrointestinal cancerBacterial composition
Escherichia coli is a widely studied model organism and an integral component of the human gut microbiome, offering significant potential for bacteria-based therapeutic applications. However, engineering native E. coli strains poses persistent challenges. In this study, the chassis- independent recombinase-assisted genome engineering technique was leveraged to engineer the native gut strain E. coli EcAZ-1 and the probiotic strain E. coli Nissle 1917 (EcN). The bioluminescent lux operon, green fluorescent protein (GFP), and the oxygen-independent fluorescent protein IFP 2.0 were successfully introduced into both strains. To further enhance IFP 2.0 fluorescence, a heme oxygenase was co-expressed, and the chromophore biliverdin was supplemented, achieving robust IFP 2.0 expression under both anaerobic and aerobic conditions. Also, both strains were engineered to biosynthesize bioactive compounds, including the plant- derived flavonoid naringenin and mycosporine-like amino acids. The results underscore the potential of native E. coli strains as flexible and robust platforms for synthetic biology, enabling novel applications in biomedical research and therapeutics. Based on these results, this application provides recombinant bacteria generating IFP2.0 fluorescence under anaerobic condition, bacterial composition for gastrointestinal cancer detection and use method thereof, and naringenin or MAA overproducing recombinant bacteria.
Owner:UNIV OF FLORIDA RESEARCH FOUNDATION INC
CRISPR-Cas system for clostridium genome engineering and recombinant strains produced thereof
A system for modifying the genome of Clostridium strains is provided based on a modified endogenous CRISPR array. The application also describes Clostridium strains modified for enhanced butanol production wherein the modified strains are produced using the novel CRISPR-Cas system.
Owner:AUBURN UNIVERSITY
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
Methods and compositions for generating polynucleotides
PCT designated stageWO2026006560A2HydrolasesVector-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
Genome engineering with CRISPR-Cas systems in eukaryotes
ActiveUS12509492B2Polypeptide with localisation/targeting motifFusion with RNA-binding domainExpression geneGenome
Disclosed herein are Type I Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) / CRISPR-associated (Cas) programmable systems and methods of using said Type I CRISPR / Cas programmable systems for activating gene expression, repressing gene expression, and gene editing. The disclosure relates to compositions that include Type I CRISPR-Cas fusion proteins designed for transcriptional activation, transcriptional repression, and / or gene editing of target genes in eukaryotic cells. The disclosure relates to Type I Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) / CRISPR-associated (Cas) transcriptional activation system related compositions and methods of using said Type I CRISPR / Cas transcriptional activation system related compositions for activating gene expression. The disclosure relates to compositions that include a Type I CRISPR-Cas fusion protein designed for transcriptional activation of target genes in eukaryotic cells.
Owner:NORTH CAROLINA STATE UNIV +1
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
Gene therapy for the treatment of activated PI3kinase delta syndrome type 1 (APDS1)
PendingUS20250288673A1Genetically modified cellsStable introduction of DNACell phenotypeGene Modification
The present invention generally relates to the field of genome engineering (gene editing), and more specifically to ex vivo gene therapy for the treatment of Activated PI3kinase Delta Syndrome type 1 (APDS1) related to PIK3CD gene. Particularly, the present invention pertains to the treatment of PIK3CD deficiency in hematopoietic stem cells (HSCs) and / or T-cells. The present invention provides means and methods for genetically modifying HSCs and / or T-cells involving gene editing reagents, such as TALE-nucleases, that specifically target an endogenous PIK3CD locus, at least in the PIK3CD allele comprising at least one APDS1-associated mutation, thereby allowing the restoration of the normal cellular phenotype. The present invention also provides engineered PIK3CD-edited HSCs and engineered PIK3CD-edited T-cells comprising at least one exogenous sequence comprising a nucleic acid sequence encoding a functional PI3Kδ protein which is integrated in said HSCs' or T-cells' genome into a PIK3CD locus, in a non-functional PIK3CD allele, resulting in the expression of a functional PI3Kδ polypeptide. The present invention further provides populations of cells comprising said engineered HSCs or T-cells, pharmaceutical compositions comprising said engineered cells or populations of cells, as well as their use in gene therapy for the treatment of APDS1.
Owner:CELLECTIS SA +4