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13 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.
Enhanced chimeric antigen receptor for immune effector cell engineering and use thereof
Provided are methods and compositions for obtaining functionally enhanced derivative effector cells obtained from the 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 use thereof comprising the functionally enhanced derivative effector cells alone, or with antibodies or checkpoint inhibitors in combination therapies.
Owner:FATE THERAPEUTICS INC
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
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
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
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
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
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
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
Genomic engineering of pluripotent cells
ActiveUS12642852B1Stable introduction of DNANucleic acid vectorInduced pluripotent stem cellGenomic engineering
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 INC