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118 results about "Genome engineering" patented technology

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.

Crispr/cas systems for genomic modification and gene modulation

The invention relates to engineered CRISPR / Cas9 systems for genomic modification and regulation of gene expression in mammalian cells. The specification describes the design and validation of polynucleotides encoding the Streptococcus pyogenes (S. pyogenes) Cas9 gene and protein and variants of that protein, where the nucleotide sequence has been optimized for expression in mammalian cells, and also modified by fused sequences that enhance various aspects of the CRISPR / Cas system. The specification also describes systems for RNA-guided genome engineering and gene regulation in mammalian cells, including human cells.
Owner:SYST BIOSCI

Meganuclease variants cleaving a DNA target sequence from the rhodopsin gene and uses thereof

The invention relates to meganuclease variants which cleave a DNA target sequence from the human Rhodopsin gene (RHO), to vectors encoding such variants, to a cell, an animal or a plant modified by such vectors and to the use of these meganuclease variants and products derived therefrom for genome therapy, ex vivo (gene cell therapy) and genome engineering including therapeutic applications and cell line engineering.
Owner:CELLECTIS SA

Meganuclease variants cleaving a DNA target sequence from the dystrophin gene and uses thereof

InactiveUS20130145487A1Expression is sufficient and stableNo impact on the expression of other genesSugar derivativesBacteriaA-DNANuclease
The invention relates to meganuclease variants which cleave a DNA target sequence from the human dystrophin gene (DMD), to vectors encoding such variants, to a cell, an animal or a plant modified by such vectors and to the use of these meganuclease variants and products derived therefrom for genome therapy, ex vivo (gene cell therapy) and genome engineering including therapeutic applications and cell line engineering. The invention also relates to the use of meganuclease variants for inserting therapeutic transgenes other than DMD at the dystrophin gene locus, using this locus as a safe harbor locus. The invention also relates to the use of meganuclease variants for using the dystrophin gene locus as a landing pad to insert and express genes of interest.
Owner:CELLECTIS SA

Compositions and methods directed to CRISPR/Cas genomic engineering systems

The invention relates to engineered CRISPR / Cas9 systems for genomic modification in mammalian cells. The present specification describes the design and testing of a polynucleotide encoding the Streptococcus pyogenes (S. pyogenes) Cas9 protein, where the nucleotide sequence has been optimized for expression in mammalian cells. The specification also describes all-in-one systems for RNA-guided genome engineering in mammalian cells, including human cells.
Owner:SYST BIOSCI

Heirarchical assembly methods for genome engineering

The present invention provides recombination based methods for assembling nucleic acids. In certain aspects the present invention provides hierarchical assembly methods for producing genome sized polynucleotide constructs. The methods may be used for assembling large polynucleotide constructs, for synthesizing synthetic genomes, or for introducing a plurality of nucleotide changes throughout the genome of an organism. In another aspect, the invention provides cells having increased genomic stability. For example, cells comprising alterations in at least a substantial portion of the transposons in the genome are provided.
Owner:CODON DEVICES

Methods for cell reprogramming and genome engineering

Methods for producing engineered induced pluripotent stem (iPS) cells are provided comprising introducing a first nucleic acid into somatic cells for integration into their genome and reprogramming the cells to produce engineered iPS cells having the nucleic acid integrated into their genome. For example, in certain aspects the cells are reprogrammed by introduction of a genetic element that expresses one or more reprogramming factor and culturing of the cells under conditions sufficient to produce reprogrammed cells.
Owner:FUJIFILM CELLULAR DYNAMICS INC

Method for modulating the efficiency of double-strand break-induced mutagenesis

A method for modulating double-strand break-induced mutagenesis at a genomic locus of interest in a cell, thereby giving new tools for genome engineering, including therapeutic applications and cell line engineering. A method for modulating double-strand break-induced mutagenesis, concerns the identification of effectors that modulate double-strand break-induced mutagenesis by use of interfering agents; these agents are capable of modulating double-strand break-induced mutagenesis through their respective direct or indirect actions on said effectors. Methods of using these effectors, interfering agents and derivatives, respectively, by introducing them into a cell in order to modulate and more particularly to increase double-strand break-induced mutagenesis. Specific derivatives of identified effectors and interfering agents, vectors encoding them, compositions and kits comprising such derivatives for modulating or increasing double-strand break-induced mutagenesis.
Owner:CELLECTIS SA

DNA nicking enzyme from a homing endonuclease that stimulates site-specific gene conversion

An engineered highly specific DNA-cleavage enzyme delivers a site-specific nick in a double stranded DNA, to cleave one DNA strand within its target site while leaving the opposing DNA strand intact. The engineered enzyme provides the ability to induce a gene conversion event in a mammalian cell. An engineered sequence-specific nickase derived from a LAGLIDADG homing endonuclease is altered by a single amino acid residue, wherein the amino acid residue is involved in the polarization of solvent molecules and acid-base catalysis in the active site without affecting direct contacts between the enzyme and either the bound DNA or bound metal ions. Engineered, site-specific nickase variants, such as of I-AniI and other homing endonucleases, are particularly useful in targeted genome engineering as well as therapeutic, targeted gene repair.
Owner:FRED HUTCHINSON CANCER RES CENT

CRISPR-Mediated Genome Engineering for Protein Depletion

The present invention provides compositions and methods for tagging a target gene with a degron (e.g., auxin-inducible degron) in a variety of eukaryotic cells using the CRISPR genome-editing technology. Also provided are cells that have been genetically modified using such compositions and methods.
Owner:THE JOHN HOPKINS UNIV SCHOOL OF MEDICINE +1

Method for increasing the efficiency of double-strand break-induced mutagenesis

InactiveUS20130337454A1Increasing double-strand break-induced mutagenesisPreventing any scarless re-ligationSugar derivativesHydrolasesGeneticsDouble stranded
The present invention relates to a method for increasing double-strand break-induced mutagenesis at a genomic locus of interest in a cell, thereby giving new tools for genome engineering, including therapeutic applications and cell line engineering. More specifically, the present invention concerns a method for increasing double-strand break-induced mutagenesis at a genomic locus of interest, leading to a loss of genetic information and preventing any scarless re-ligation of said genomic locus of interest by NHEJ. The present invention also relates to engineered endonucleases, chimeric or not, vectors, compositions and kits used to implement this method.
Owner:CELLECTIS SA

Rna-guided gene editing and gene regulation

Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) / CRISPR-associated (Cas) 9-based system related compositions and methods of using said CRISPR / Cas9-based system related compositions for altering gene expression and genome engineering. Also disclosed herein are compositions and methods of using said compositions for altering gene expression and genome engineering in muscle, such as skeletal muscle and cardiac muscle.
Owner:DUKE UNIV

Genomic engineering of pluripotent cells

Provided are methods and compositions for obtaining genome-engineered iPSCs, and derivative cells with stable and functional genome editing at selected sites. Also provided are cell populations or clonal cell lines derived from genome-engineered iPSCs, which comprise targeted integration of one or more exogenous polynucleotides, and / or in / dels in one or more selected endogenous genes.
Owner:FATE THERAPEUTICS

Novel crispr-associated (CAS) protein

ActiveUS20180282715A1Facilitates target knockdownHydrolasesClimate change adaptationSingle-Stranded RNANucleotide
A new CRISPR-associated (Cas) protein, termed “CasM,” is described, as well as polynucleotides encoding the same and methods of using CasM for site-specific genome engineering. CasM proteins are capable of targeting and cleaving single-stranded RNA.
Owner:LOCANABIO INC

Rationally-designed meganucleases for maize genome engineering

ActiveUS20110113509A1HydrolasesPeptide preparation methodsBiotechnologyDna recognition
The invention relates to the field of molecular biology and recombinant nucleic acid technology. In particular, the invention relates to a rationally-designed, non-naturally-occurring meganuclease with altered DNA recognition sequence specificity which recognizes and cleaves a unique DNA site in the maize genome. The invention also relates to methods of producing engineered maize plants using such meganucleases.
Owner:PRECISION BIOSCI

Genomic engineering of pluripotent cells

Provided are methods and compositions for obtaining genome-engineered iPSCs, and derivative cells with stable and functional genome editing at selected sites. Also provided are cell populations or clonal cell lines derived from genome-engineered iPSCs, which comprise targeted integration of one or more exogenous polynucleotides, and / or in / dels in one or more selected endogenous genes.
Owner:FATE THERAPEUTICS

Cas9 nuclease platform for microalgae genome engineering

The present invention relates to a method of genome engineering in microalgae using the Cas9 / CRISPR system. In particular, the present invention relates to methods of delivering RNA guides via cell penetrating peptides in microalgae, preferably in stable integrated Cas9 microalgae. The present invention also relates to kits and isolated cells comprising Cas9, split Cas9 or guide RNA and Cas9-fused cell-penetrating peptides. The present invention also relates to isolated cells obtained by the methods of the invention.
Owner:CELLECTIS SA

Variants of cpf1 (cas12a) with altered pam specificity

Engineered CRISPR from Prevotella and Francisella 1 (Cpf1) nucleases with improved targeting range and enhanced on-target activity, and their use in genomic engineering, epigenomic engineering, base editing, genome targeting, genome editing, and in vitro diagnostics.
Owner:THE GENERAL HOSPITAL CORP

IMMUNOTHERAPIES USING ENHANCED iPSC DERIVED EFFECTOR CELLS

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

Method for increasing the efficiency of double-strand-break induced mutagenesis

The present invention relates to a method for increasing double-strand-break induced mutagenesis at a genomic locus of interest in a cell, thereby giving new tools for genome engineering, including therapeutic applications and cell line engineering. More specifically, the present invention concerns the combined use of TALEN or meganucleases with TREX2, especially under the form of single-chain proteins.
Owner:CELLECTIS SA

Novel mitochondrial genome editing tool

The present invention discloses a novel mitochondrial genome editing tool, and belongs to the field of genome engineering. According to the prevent invention, the constructed mtCRISPR / Cas9 system mainly comprises two parts such as gRNA entering mitochondria and Cas9 nuclease localized in mitochondria, wherein the constructed mt-gRNA has two forms, the one mt-gRNA comprises a RNA mitochondrial localizing guide sequence, a targeting sequence and a gRNA skeleton sequence, the other mt-gRNA comprises a RNA mitochondrial localizing guide sequence, any one tRNA sequence encoded by mitochondrial or other additional spacer sequences, a targeting sequence and a gRNA skeleton sequence, the obtained combined material acts on the mitochondrial genome to break the target sequence in a targeted manner after the mt-gRNA and the mtCas9 nuclease are combined, and the action efficiency of the second mt-gRNA action is high than the action efficiency of the mt-gRNA; and the results verify that the constructed mtCRISPR / Cas9 system has characteristics of high efficiency and strong specificity.
Owner:聂凌云

Method for increasing the efficiency of double-strand-break induced mutagenesis

The present invention relates to a method for increasing double-strand-break induced mutagenesis at a genomic locus of interest in a cell, thereby giving new tools for genome engineering, including therapeutic applications and cell line engineering. More specifically, the present invention concerns the combined use of TALEN or meganucleases with TREX2, especially under the form of single-chain proteins.
Owner:CELLECTIS SA

I-crei meganuclease variants with modified specificity, method of preparation and uses thereof

Method of preparing I-CreI meganuclease variants having a modified cleavage specificity, variants obtainable by said method and their applications either for cleaving new DNA target or for genetic engineering and genome engineering for non-therapeutic purposes. Nucleic acids encoding said variants, expression cassettes comprising said nucleic acids, vectors comprising said expression cassettes, cells or organisms, plants or animals except humans, transformed by said vectors.
Owner:CELLECTIS SA

Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy

Disclosed herein are vectors that targets a dystrophin gene, encoding at least one Cas9 molecule or a Cas9 fusion protein, and at least one gRNA molecule (e.g., two gRNA molecules), and compositions and cells comprising such vectors. Also provided are methods for using the vectors, compositions and cells for genome engineering (e.g., correcting a mutant dystrophin gene), and for treating DMD.
Owner:EDITAS MEDICINE +1
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