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27 results about "Meganuclease" patented technology
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Meganucleases are endodeoxyribonucleases characterized by a large recognition site (double-stranded DNA sequences of 12 to 40 base pairs); as a result this site generally occurs only once in any given genome. For example, the 18-base pair sequence recognized by the I-SceI meganuclease would on average require a genome twenty times the size of the human genome to be found once by chance (although sequences with a single mismatch occur about three times per human-sized genome). Meganucleases are therefore considered to be the most specific naturally occurring restriction enzymes.
Methods and compositions for treating hepatitis b virus-related conditions
The present disclosure encompasses a lipid nanoparticle (LNP) comprising a polypeptide comprising a nucleic acid sequence encoding an engineered meganuclease that binds and cleaves a recognition sequence within a Hepatitis B virus (HBV) genome. Further, the disclosure encompasses pharmaceutical compositions comprising the LNPs, and the use of such compositions for inactivating a pol gene of an HBV genome or an HBV genome fragment in a cell and treating HBV infections or diseases associated with HBV infections.
Owner:PRECISION BIOSCIENCES INC +1
Treatment of HSV-2 using megasnuclease
Described herein are compositions and methods for reducing or eliminating latent herpes simplex virus type 2 (HSV-2) in HSV-2 infected cells, or for reducing or eliminating latent HSV-2 reactivation in HSV-2 infected cells, thereby providing viable therapeutic methods for latent HSV-2 infection. The compositions comprise a plurality of viral vectors, where the viral vectors comprise a sequence encoding a HSV-2 specific megasuclease. The method comprises delivering a plurality of one or more viral vectors to the HSV-2 infected cell, where each of the one or more viral vectors comprises a sequence encoding an HSV-2 specific megasuclease.
Owner:FRED HUTCHINSON CANCER RESEARCH CENTER
Engineered meganucleases that target human mitochondrial genomes
Disclosed herein are engineered meganucleases engineered to recognize and cleave a recognition sequence present in human mitochondrial DNA (mtDNA). The disclosure further relates to the use of such engineered meganucleases in methods for producing genetically-modified eukaryotic cells, and to a population of genetically-modified eukaryotic cells, wherein mutant mtDNA is modified or degraded.
Owner:UNIV OF MIAMI +1
Modified cells and therapeutic methods
The present invention provides a genetically modified composition for treating cancer, and a method for preparing and using a genetically modified composition in the treatment of cancer. [Solution] The compositions and methods disclosed herein can be used to identify cancer-specific T cell receptors (TCRs) that recognize unique immunogenic mutations in a patient's cancer and to treat any type of cancer in the patient. Insertion of these transgenes encoding cancer-specific TCRs into T cells, using non-viral methods (e.g., CRISPR, TALEN, transposon-based ZEN, meganuclease, or Mega-TAL), is a novel technique that opens up new opportunities to extend immunotherapy to many cancer types.
Owner:REGENTS OF THE UNIVERSITY OF MINNESOTA +2
Treatment of retinitis pigmentosa using improved engineered meganucleases
Disclosed are recombinant meganucleases engineered to bind and cleave a recognition sequence present in a mutant RHO P23H allele. The invention further relates to the use of such recombinant meganucleases in a method for treating retinitis pigmentosa, wherein the mutant RHO P23H allele is preferentially targeted, cleaved, and inactivated.
Owner:PRECISION BIOSCIENCES INC
MANIPULATED MEGANUCLEASES AGAINST HUMAN MITOCHONDRIAL GENOMES
Owner:PRECISION BIOSCIENCES INC +1
Engineered meganucleases specific for recognition sequences in the dystrophin gene
This disclosure covers engineered macronucleases that bind to and cleave recognition sequences within the dystrophin gene. This disclosure also covers methods for preparing genetically modified cells using such engineered macronucleases. Furthermore, this disclosure covers pharmaceutical compositions comprising engineered macronuclease proteins or polynucleotides encoding engineered macronucleases of this disclosure, and the use of such compositions for modifying the dystrophin gene in a subject or for treating Duchenne muscular dystrophy.
Owner:PRECISION BIOSCIENCES INC
Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
Rationally-designed LAGLIDADG meganucleases and methods of making such meganucleases are provided. In addition, methods are provided for using the meganucleases to generate recombinant cells and organisms having a desired DNA sequence inserted into a limited number of loci within the genome, as well as methods of gene therapy, for treatment of pathogenic infections, and for in vitro applications in diagnostics and research.
Owner:DUKE UNIV
Engineered meganucleases having specificity for a recognition sequence in the hepatitis b virus genome
Owner:PRECISION BIOSCIENCES INC
MANIPULATED MEGANUCLEASES WITH SPECIFICATION FOR DETECTION SEQUALS IN THE DYSTROPHING GENE
Owner:PRECISION BIOSCIENCES INC
Optimized engineered meganucleases having specificity for the human t cell receptor alpha constant region gene
The present invention encompasses engineered meganucleases that bind and cleave a recognition sequence within the first exon of the human T cell receptor (TCR) alpha constant region gene. The engineered meganucleases can exhibit at least one optimized characteristic, such as increased specificity or efficiency of cleavage, when compared to previous generations of meganucleases. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells, and the use of such cells in a pharmaceutical composition and in methods for treating diseases, such as cancer.
Owner:PRECISION BIOSCIENCES INC
Optimized engineered nucleases specific for the human t cell receptor alpha constant region gene
ActiveCN112218889BPolypeptide with localisation/targeting motifImmunoglobulin superfamilyExonT-cell receptor alpha constant
The invention includes engineered nucleases that recognize and cleave a recognition sequence within the first exon of the human T cell receptor (TCR) alpha constant region gene. The engineered meganucleases can exhibit at least one optimized property, such as enhanced (i.e., increased) specificity or cleavage efficiency, as compared to the first generation meganuclease TRC 1-2x.87EE. The invention also includes methods of using such engineered nucleases to make genetically modified cells, as well as uses of such cells in pharmaceutical compositions and in methods of treating diseases such as cancer.
Owner:PRECISION BIOSCIENCES INC
Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
Owner:PRECISION BIOSCIENCES INC
Polypeptide linkers for use in engineered meganucleases
Owner:PRECISION BIOSCIENCES INC
Engineered meganucleases having specificity for recognition sequences in the c9ORF72 gene
PCT designated stageWO2026028165A1Nervous disorderVectorsAmyotrophic lateral sclerosisPharmaceutical drug
The present disclosure encompasses engineered meganucleases that bind and cleave recognition sequences within a C9Orf72 gene. The present disclosure also encompasses methods of using such engineered meganucleases to make genetically modified cells. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or polynucleotides encoding engineered meganucleases of the disclosure, and the use of such compositions for the modification of a C9Orf72 gene in a subject, or for treatment of amyotrophic lateral sclerosis (ALS) and / or frontotemporal dementia (FTD).
Owner:PRECISION BIOSCIENCES INC
Optimized engineered meganucleases having specificity for a recognition sequence in the hepatitis b virus genome
The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within a Hepatitis B virus (HBV) genome. The engineered meganucleases can exhibit at least one optimized characteristic, such as enhanced specificity and / or efficiency of indel formation, when compared to previously described HBV meganucleases. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or hepatocellular carcinoma.
Owner:PRECISION BIOSCIENCES INC
Polypeptide linkers for use in engineered meganucleases
The present disclosure encompasses engineered meganucleases that comprise two subunits connected by a polypeptide linker that is smaller than polypeptide linkers used in the art. The engineered meganucleases can exhibit at least one improved characteristic, such as increased efficiency of binding and / or cleavage, when compared to meganucleases comprising longer polypeptide linkers. The present disclosure also encompasses methods of using engineered nucleases to make genetically-modified cells and the use of such cells in a pharmaceutical composition and in methods for treating diseases.
Owner:PRECISION BIOSCIENCES INC
Fusion molecules of rationally-designed DNA-binding proteins and effector domains
PendingUS20250346874A1Polypeptide with localisation/targeting motifFusion with DNA-binding domainTranscription RepressorBinding domain
Targeted transcriptional effectors (transcription activators and transcription repressors) derived from meganucleases are described. Also described are nucleic acids encoding same, and methods of using same to regulate gene expression. The targeted transcriptional effectors can comprise (i) a meganuclease DNA-binding domain lacking endonuclease cleavage activity that binds to a target recognition site; and (ii) a transcription effector domain.
Owner:DUKE UNIV
Genetically modified t cells comprising a modified intron of the t cell receptor alpha gene
To provide engineered meganucleases.SOLUTION: Provided is an engineered meganuclease that recognizes and cleaves a recognition sequence within an intron of the human T cell receptor alpha gene located 5 ' upstream of TRAC exon 1, wherein said engineered meganuclease comprises a first subunit and a second subunit, wherein said first subunit binds to a first recognition half-site of said recognition sequence and comprises a first hypervariable region, and wherein said second subunit binds to a second recognition half-site of said recognition sequence and comprises a second hypervariable region.SELECTED DRAWING: None
Owner:PRECISION BIOSCIENCES INC
Muscle-specific expression cassettes
The present disclosure encompasses nucleic acid expression constructs that regulate the expression of a heterologous protein specifically in muscle tissues (e.g., skeletal muscle and cardiac muscle tissue). In particular embodiments of the disclosure, muscle-specific expression constructs are described that encode a heterologous protein. These heterologous proteins can be any protein that is desired to be expressed in a muscle cell. In particular embodiments of the disclosure, proteins are described for the treatment of Duchenne Muscular Dystrophy (e.g., engineered nucleases such as engineered meganucleases).
Owner:PRECISION BIOSCIENCES INC
Treatment of HSV-2 using meganuclease
Described herein are compositions and methods for providing a viable therapeutic approach to latent herpes simplex virus type 2 (HSV-2) infection by reducing or eliminating latent HSV-2 from HSV-2-infected cells, or by reducing or eliminating the reactivation of latent HSV-2 in HSV-2-infected cells. The compositions comprise a plurality of viral vectors, each comprising a sequence encoding an HSV-2-specific meganuclease. The methods comprise the step of delivering a plurality of viral vectors to HSV-2-infected cells, each of which comprises a sequence encoding an HSV-2-specific meganuclease.
Owner:FRED HUTCHINSON CANCER RESEARCH CENTER
Engineered meganucleases having specificity for a recognition sequence in the hepatitis b virus genome
ActiveUS20260028603A1Polypeptide with localisation/targeting motifPeptide/protein ingredientsHepatitis B immunizationPharmaceutical drug
The present disclosure encompasses engineered nucleases which recognize and cleave a recognition sequence within a Hepatitis B virus (HBV) genome. The engineered meganucleases described herein can exhibit improved characteristics, such as enhanced specificity and / or efficiency of indel formation, when compared to previously described HBV meganucleases. Further, the disclosure encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating HBV infections or diseases associated with HBV infections.
Owner:PRECISION BIOSCIENCES INC
Genetically modified meganucleases that target the human mitochondrial genome
The present disclosure provides compounds of formula (II), where R1 is an optionally substituted monocyclic or bicyclic saturated, partially unsaturated or aromatic heterocyclyl containing at least one nitrogen atom, and other variables are as defined herein. The compounds are intended to inhibit the activity of coronavirus main protease (M PRO ). Methods for the preparation of the compounds and uses of the compounds, for example in the treatment and / or prevention of coronavirus diseases, such as COVID-19, are also disclosed. [Formula 1] TIFF2024514939000027.tif26170
Owner:PRECISION BIOSCIENCES INC +1
Polypeptide linkers for use in engineered meganucleases
The present disclosure encompasses engineered meganucleases that comprise two subunits connected by a polypeptide linker that is smaller than polypeptide linkers used in the art. The engineered meganucleases can exhibit at least one improved characteristic, such as increased efficiency of binding and / or cleavage, when compared to meganucleases comprising longer polypeptide linkers. The present disclosure also encompasses methods of using engineered nucleases to make genetically-modified cells and the use of such cells in a pharmaceutical composition and in methods for treating diseases.
Owner:PRECISION BIOSCIENCES INC
MANIPULATED MEGANUCLEASES WITH SPECIFICATION FOR A RECOGNITION SEQUENCE IN THE TRANSTHYRETIN GENE
Owner:PRECISION BIOSCIENCES INC
Compositions and methods for targeting donor polynucelotides in soybean genomic loci
Compositions and methods are provided for soybean genomic integration sites. These methods and compositions for producing a soybean plant comprise targeting the soybean genomic integration site with a site specific nuclease. The use of site specific nucleases for creating a double-strand-break target site can be performed with a zinc finger endonuclease, an engineered endonuclease, a meganuclease, a TALENs and / or a CRISPRCas endonuclease. The soybean genomic integration site can comprise at least one genomic locus of interest such as a trait cassette, a transgene, a mutated gene, a native gene, an edited gene or a site-specific integration (SSI) target site.
Owner:PIONEER HI BREED INTERNATIONAL INC
RATIONALLY DESIGNED SINGLE-CHAIN ​​MEGANUCLEASES WITH NON-PALINDROME DETECTION SEQUALS
Owner:PRECISION BIOSCIENCES INC