Patents Issued in August 31, 2023
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Publication number: 20230272342Abstract: The present invention relates to regulatory T cell and uses thereof. By their immunosuppressive and anti-inflammatory activities, regulatory T cells play a central role in peripheral tolerance and thus critically prevent the development of autoimmune and inflammatory disorders. The inventors showed that Foxp3+CD4+ Tregs express high levels of LT?, which negatively regulates their immunosuppressive signature. They demonstrated that the adoptive transfer of LT??/? Tregs in mice protects from dextran sodium sulfate (DSS)-induced colitis and attenuates inflammatory bowel disease (IBD), multi-organ autoimmunity and the development of CAC. The inventors also showed that by mixed bone marrow chimeras that LT? expression specifically in hematopoietic cells negatively controls the immunosuppressive signature of Tregs. In particular, the present invention relates to regulatory T cell characterized in that it does not express or expresses reduced levels of lymphotoxin alpha.Type: ApplicationFiled: February 13, 2023Publication date: August 31, 2023Inventor: Magali IRLA
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Publication number: 20230272343Abstract: An artificial antigen presenting cell system comprising one or more gelated human dendritic cells and a controlled release system capable of releasing one or more cytokines. Also provided herein are methods for producing the gelated human dendritic cells and uses of the artificial antigen presenting cell system for activating immune cells.Type: ApplicationFiled: May 7, 2021Publication date: August 31, 2023Applicant: Celtec, Inc.Inventors: Che-Ming Jack Hu, Jung-Chen Lin, Chung-Yao Hsu
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Publication number: 20230272344Abstract: The methods of the invention employ targeted magnetic particles, preferably targeted nanomagnetic particles, and targeted buoyant particles such as buoyant microparticles and microbubbles. Among the benefits of the invention is the ability to combine targeted magnetic particles with differentially targeted buoyant particles to achieve separation of two or more specifically cell targeted populations during the same work flow.Type: ApplicationFiled: January 19, 2023Publication date: August 31, 2023Applicant: BIOLEGEND, INC.Inventors: DHANESH GOHEL, HONG ZHANG, JOHN RANSOM
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Publication number: 20230272345Abstract: This application relates to a method of making a cell construct, comprising a) plating a plurality of cells on a substantially flat surface; b) growing the plurality of cells to at least 80% confluent to form a cell sheet with intercellular linkages; c) applying a culture medium having a pH of about 5 to about 6.8 to the cell sheet; d) replacing the culture medium of step c) with a culture medium having a pH of about 7.5 to about 8.5; and e) replacing the culture medium of step d) with a culture medium having a pH of about 7 to about 7.7, to obtain a substantially planar untethered cell sheet. Also provided is a cell construct formed according to the method and uses thereof.Type: ApplicationFiled: June 30, 2021Publication date: August 31, 2023Inventors: Ponnambalam Ravi Selvaganapathy, Alireza Shahin-Shamsabadi
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Publication number: 20230272346Abstract: Provided herein are methods to increase the culture density and/or thickness of a cellular biomass in a cultivation infrastructure, to improve the culture of cells in the absence of serum in a cultivation infrastructure, and to promote anchorage-independent growth of a cellular biomass in a cultivation infrastructure. The methods comprise inhibiting the HIPPO signaling pathway, for example, by activating YAP1, activating TAZ, and/or inhibiting MOB1, LATS1 kinase, LATS2 kinase, WW45, MST1 kinase, and/or MST2 kinase in the cellular biomass. In some embodiments, the cellular biomass is harvested from the cultivation infrastructure for the formulation of cell-based food products or ingredients, such as animal meat manufactured from cells in an ex vivo process or for therapeutic applications such as organ or tissue transplantation or grafting.Type: ApplicationFiled: October 12, 2022Publication date: August 31, 2023Inventors: Nicholas J. GENOVESE, Meri Teresa FIRPO, Daphné DAMBOURNET
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Publication number: 20230272347Abstract: The invention relates to differentiation methods for progenitor cells, e.g. mammalian epithelial stem cells, differentiation media for use in said methods, organoids and cells obtainable by said methods and uses, including therapeutic uses, thereof.Type: ApplicationFiled: January 23, 2023Publication date: August 31, 2023Applicant: Koninklijke Nederlandse Akademie Van WetenschappenInventors: Johannes Carolus Clevers, Helmuth Gehart
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Publication number: 20230272348Abstract: The present invention relates to a method of producing an enriched preparation of hemogenic endothelial progenitor cells. This method involves providing a population of pluripotent stem cells and inducing expression of a SOXF transcription factor in the pluripotent stem cells of the population. The method further involves culturing the population of pluripotent stem cells expressing the SOXF transcription factor, whereby the enriched preparation of hemogenic endothelial progenitor cells is produced as a result of said culturing. The present invention also relates to a method of producing an enriched preparation of hematopoietic progenitor cells and methods of treating a subject having a condition mediated by a loss or dysfunction of hematopoietic stem cells or by a loss of immune cells.Type: ApplicationFiled: June 22, 2021Publication date: August 31, 2023Inventors: Xiaojun Lian, Lauren Nicole Randolph, Yuqian Jiang
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Publication number: 20230272349Abstract: Modified E1a regulatory sequences are provided, wherein at least one Pea3 binding site, or a functional portion thereof, is deleted. Also provided are modified E1a sequences that selectively express particular isoforms. Also provided is an E1b-19K clone insertion site. These modified sequences can be used individually, or in combination with one another, to provide tumor-selective expression of proteins.Type: ApplicationFiled: November 23, 2022Publication date: August 31, 2023Inventors: Tony REID, Farah HEDJRAN, Shantanu KUMAR
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Publication number: 20230272350Abstract: The present invention relates to a method of purifying whole HCV particles, the method comprising the steps of a) providing a cell culture supernatant comprising virus particles, b) purification and/or concentration of the cell culture supernatant, c) purification and/or concentration of the product of above step b) using steric exclusion chromatography (SXC) at alkaline pH in the range of 8-10, d) purification and/or concentration of the product of above step c) using sulphated cellulose membrane absorbers (SCMA), e) obtaining purified whole virus particles.Type: ApplicationFiled: July 12, 2021Publication date: August 31, 2023Inventors: Judith Margarete Gottwein, Jens Bukh, Anna Offersgaard, Anne Finne Pihl, Michael Wolff, Keven Lothert
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Publication number: 20230272351Abstract: Provided herein is a synthetic viral particle that positive control for COVID assays, along with methods for making and using the viral particle.Type: ApplicationFiled: September 1, 2021Publication date: August 31, 2023Inventors: Nicole Steinmetz, Soo Khim Chan
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Publication number: 20230272352Abstract: The present invention relates to a bacteriophage composition comprising one or more (suitably two or more, or three) bacteriophages selected from Sa87, J-Sa36, or Sa83, or mutants thereof. Also encompassed are the use of said bacteriophage compositions for medical or non-medical applications, as well as kits, bandages, and wound dressings comprising said bacteriophage compositions.Type: ApplicationFiled: October 25, 2022Publication date: August 31, 2023Inventors: Karen Joy SHAW, Sandra P. MORALES, Gillian MEARNS, Deborah A. RANKIN, Frenk SMREKAR
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Publication number: 20230272353Abstract: The instant invention relates to a filamentous fungal host cell producing a polypeptide of interest, said host cell comprising one or more native or heterologous polynucleotide encoding a long non-coding RNA (lncRNA), wherein said lncRNA comprises more than 262 nucleotides and comprises a contiguous nucleotide sequence at its 3? end, absent any poly-adenylation tail, that is at least 70% identical to that of SEQ ID NO:8, SEQ ID NO:9, SEQ ID NO: 10, SEQ ID NO: 19, SEQ ID NO:20 or SEQ ID NO:21; and/or said lncRNA comprises 2 or more Xyr1 binding sequences, as well as methods of producing a polypeptide of interest in said host cells and methods of improving the production of a polypeptide of interest.Type: ApplicationFiled: February 21, 2019Publication date: August 31, 2023Applicant: Novozymes A/SInventors: Debbie Yaver, Astrid Mach-Aigner, Robert Mach, Petra Till
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Publication number: 20230272354Abstract: The present invention provides engineered proline hydroxylase polypeptides for the production of hydroxylated compounds, polynucleotides encoding the engineered proline hydroxylases, host cells capable of expressing the engineered proline hydroxylases, and methods of using the engineered proline hydroxylases to prepare compounds useful in the production of active pharmaceutical agents.Type: ApplicationFiled: March 8, 2023Publication date: August 31, 2023Inventors: Jovana Nazor, Robert Osborne, Jack Liang, Jonathan Vroom, Xiyun Zhang, David Entwistle, Rama Voladri, Ravi David Garcia, Jeffrey C. Moore, Shane Grosser, Birgit Kosjek, Matthew Truppo
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Publication number: 20230272355Abstract: This disclosure provides various SPIN transposases and transposons, systems, and methods of use.Type: ApplicationFiled: June 4, 2021Publication date: August 31, 2023Inventors: David Largaespada, Branden Moriarity, Beau Webber, Neil Otto, Sandeep Kumar, Bryan Jones
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Publication number: 20230272356Abstract: The disclosure provides Moloney murine leukemia virus (MMLV) reverse transcriptase (RTase) mutants with C-terminal peptide and/or N-terminal peptide extensions that improve the performance of the MMLV RTase mutants. The disclosure also provides suitable amino acid positions in MMLV RTases for mutagenesis and methods and kits for using MMLV RTase mutants to synthesize cDNA from RNA templates.Type: ApplicationFiled: February 28, 2023Publication date: August 31, 2023Inventors: Sarah Franz Beaudoin, Tanner Holden Reeb, Christopher Anthony Vakulskas
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Publication number: 20230272357Abstract: Described herein is an ex vivo method of site-specifically editing a target cell genome, the method including treating a population of unmodified target cells with a Class I and/or Class II histone deacetylase inhibitor to provide a population of chromatin decondensed unmodified target cells; and introducing into the population of chromatin decondensed unmodified target cells a Cas9 ribonucleoprotein, to provide a population of site-specifically genome-edited target cells; wherein the Cas9 ribonucleoprotein comprises a Cas9 protein and a guide RNA and cleaves DNA at a cleavage site in the target cell genome.Type: ApplicationFiled: November 4, 2022Publication date: August 31, 2023Inventors: Krishanu Saha, Kaivalya Molugu
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Publication number: 20230272358Abstract: The present invention relates to a Bacillus host cell belonging to the species Bacillus lichemformis or Bacillus pumilus in which the chromosomal alr gene has been inactivated. Said bacterial host cell comprises a plasmid comprising at least one autonomous replication sequence, a first polynucleotide encoding at least one polypeptide of interest, wherein said first polynucleotide is operably linked to a promoter, and a second polynucleotide encoding an alanine racemase which is not native to the host cell, wherein said second polynucleotide is operably linked to a promoter. The present invention further relates to a method for producing at least one polypeptide of interest based on cultivating the bacterial host cell of the present invention.Type: ApplicationFiled: July 23, 2021Publication date: August 31, 2023Inventors: Stefan Jenewein, Max Fabian Felle, Christopher Sauer, Tobias Klein
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Publication number: 20230272359Abstract: The present invention relates, in part, to compositions and methods for treating or preventing coronavirus infection.Type: ApplicationFiled: July 20, 2021Publication date: August 31, 2023Inventors: James Torchia, Gordon J. Freeman
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RECOMBINANT VITAMIN K DEPENDENT PROTEINS WITH HIGH SIALIC ACID CONTENT AND METHODS OF PREPARING SAME
Publication number: 20230272360Abstract: Methods of isolating highly sialylated recombinant vitamin K dependent proteins, particularly Factor IX, by chromatographic methods am described. The highly sialylated recombinant proteins are characterized. The improved Factor IX has at least 62% N-glycosylation with 3 or 4 sialic acid residues and improved bioavailability and pharmokinetic properties.Type: ApplicationFiled: December 10, 2021Publication date: August 31, 2023Inventors: Michael J. Griffith, William N. Drohan -
Publication number: 20230272361Abstract: The invention is mainly in the field of the production of food and luxury foods, for example coffee and coffee substitute products. Enzymes are provided which are capable of degrading acrylamide—preferably also at temperatures above 50° C., in particular in temperature ranges which occur in the production of coffee/coffee substitute products, and/or at a pH value between pH 4 and pH 7, as is usual in the production of coffee/coffee substitute products. Furthermore, methods for degrading acrylamide from preparations selected from semi-finished goods as well as finished goods are provided. Also, the present invention relates to preparations having a reduced acrylamide content compared to preparations which have not been subjected to the method for removing acrylamide according to the invention by means of the enzymes according to the invention.Type: ApplicationFiled: January 21, 2021Publication date: August 31, 2023Inventors: Oliver SÜSSE-HERRMANN, Sabrina KÖPKE, Andreas VOGEL, Claudia FELLER, Sebastian BARTSCH
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Publication number: 20230272362Abstract: The invention is mainly in the field of the production of food and luxury foods, for example coffee and coffee substitute products. Enzymes are provided which are capable of degrading acrylamide - preferably also at temperatures above 50° C., in particular in temperature ranges which occur in the production of coffee/coffee substitute products, and/or at a pH value between pH 4 and pH 7, as is usual in the production of coffee/coffee substitute products. Furthermore, methods for degrading acrylamide from preparations selected from semi-finished goods as well as finished goods are provided. Also, the present invention relates to preparations having a reduced acrylamide content compared to preparations which have not been subjected to the method for removing acrylamide according to the invention by means of the enzymes according to the invention.Type: ApplicationFiled: January 21, 2021Publication date: August 31, 2023Inventors: Oliver SÜSSE-HERRMANN, Sabrina KÖPKE, Andreas VOGEL, Claudia FELLER, Sebastian BARTSCH
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Publication number: 20230272363Abstract: The present invention provides engineered penicillin G acylase (PGA) enzymes, polynucleotides encoding the enzymes, compositions comprising the enzymes, and methods of using the engineered PGA enzymes.Type: ApplicationFiled: March 23, 2023Publication date: August 31, 2023Inventors: Rama Voladri, Christopher Michael Micklitsch, Oscar Alvizo, Jovana Nazor, Da Duan, Judy Victoria Antonio Viduya, Stephan Jenne, Chihui An, Keith Allen Canada, Paul N. Devine, Iman Farasat, Anna Fryszkowska, Katrina W. Lexa, Robert Kevin Orr
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Publication number: 20230272364Abstract: Abstract: Gluconate dehydratase enzymes and recombinant cells are provided, along with their use in the production of 2-ke-to-3-deoxy-D-gluconate (KDG).Type: ApplicationFiled: July 13, 2020Publication date: August 31, 2023Applicant: BP Corporation North America Inc.Inventor: Fernando Valle
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Publication number: 20230272365Abstract: The invention relates to archaeal pyrrolysyl tRNA synthetases lacking a nuclear localization signal and/or comprising a nuclear export signal. The invention also relates to polynucleotides encoding said pyrrolysyl tRNA synthetases, eukaryotic cells comprising said polynucleotide and tRNA acylated by the pyrrolysyl tRNA synthetase or a polynucleotide encoding such tRNA, methods utilizing said cells for preparing polypeptides comprising unnatural amino acid residues, and kits useful in said methods.Type: ApplicationFiled: September 27, 2022Publication date: August 31, 2023Applicant: European Molecular Biology LaboratoryInventors: Edward Lemke, Ivana Nikic, Gemma Estrada Girona, Christine Köhler
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Publication number: 20230272366Abstract: Disclosed are a mutant of a pyruvate carboxylase gene promoter of Corynebacterium glutamicum and applications thereof. The mutant has improved promoter activity compared with a wild-type promoter, and can be used for enhancing expression of a target gene, for example, operably ligating the mutant to a pyruvate carboxylase gene enhances the expression intensity of the pyruvate carboxylase, thereby improving the production efficiency of amino acids of the strain.Type: ApplicationFiled: July 13, 2021Publication date: August 31, 2023Inventors: Jibin Sun, Jiao Liu, Ping Zheng, Tuo Shi, Wenjuan Zhou, Jiuzhou Chen, Xuan Guo, Yanhe Ma
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Publication number: 20230272367Abstract: In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production.Type: ApplicationFiled: May 12, 2023Publication date: August 31, 2023Applicant: PTC THERAPEUTICS, INC.Inventors: Anuradha Bhattacharyya, Amal Dakka, Kerstin Effenberger, Vijayalakshmi Gabbeta, Wencheng Li, Nikolai Naryshkin, Christopher Trotta, Kari Wiedinger
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Publication number: 20230272368Abstract: Disclosed are nucleic acid preserving compositions and methods of manufacturing and using the same, particularly for detection of COVID-19 virus. Compositions include a carrier, a chaotropic agent, a buffering agent, a chelating agent, a surfactant, an alcohol, an acid, and a mucolytic agent. Compositions as aqueous solutions can include water as a carrier. Preferred embodiments include water, guanidine thiocyanate, Tris, EDTA, SLS, SDA 3C, HCl, and N-acetyl-L-cysteine. Some embodiments include a colored dye as a visual indicator. Kits include the composition disposed in a portion of a biological sample collection apparatus. Methods of manufacturing include combining the components into a mixture, such as an aqueous solution. Methods of use include providing a biological sample that includes nucleic acid and contacting the biological sample with the composition. Detection of COVID-19 virus is demonstrated. Compositions also preserve and stabilize human nucleic acid for subsequent analysis.Type: ApplicationFiled: April 14, 2021Publication date: August 31, 2023Inventors: Federico Carlos Arejola Gaeta, William Phillips
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Publication number: 20230272369Abstract: The present invention addresses the problem of providing a novel method which is for preparing a DNA fragment for microbial cell transformation, and by which the combinatorial library of a long-chain DNA can be efficiently constructed and confirmation of the genotype of the obtained clone is facilitated.Type: ApplicationFiled: March 24, 2023Publication date: August 31, 2023Inventors: Kenji TSUGE, Jun ISHII, Akihiko KONDO
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Publication number: 20230272370Abstract: Provided is a product, composition that contains genetically encoded YEATS domain probe. Provided is a method of making the genetically encoded YEATS domain probes. Also provided are methods of modulating YEATS domain proteins using the genetically encoded probes.Type: ApplicationFiled: July 27, 2021Publication date: August 31, 2023Inventors: Xiang David LI, Xin LI, Gaofei TIAN
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Publication number: 20230272371Abstract: A method for screening for a polypeptide that acts on a target protein, including (1) providing a polynucleotide library constituted of a plurality of expression vectors that can be expressed in a gram-negative bacterium, where the plurality of expression vectors each include a first polynucleotide encoding a polypeptide different from one another, a secretory signal sequence positioned upstream of the first polynucleotide, and a second polynucleotide encoding a target protein, (2) transforming a gram-negative bacterium with the expression vector to express the polypeptide in a periplasmic space and the target protein on an inner membrane surface, (3) contacting the polypeptide with the target protein in the periplasmic space, (4) allowing the gram-negative bacterium to form a spheroplast to measure activity of the polypeptide on the target protein by a patch clamp technique, and (5) identifying the polypeptide that acts on the target protein based on the measured activity.Type: ApplicationFiled: August 4, 2021Publication date: August 31, 2023Applicant: NATIONAL INSTITUTE OF ADVANCED INDUSTRIAL SCIENCE AND TECHNOLOGYInventor: Tadashi KIMURA
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Publication number: 20230272372Abstract: A cellular coding construct uniquely codes a cellular entity and includes a laser particle and a structurally coded oligonucleotide. The structurally coded oligonucleotide and the laser particle have a physical association with each other and are configured for physical association with the cellular entity and also configured for distinctive identification of the cellular entity.Type: ApplicationFiled: August 17, 2022Publication date: August 31, 2023Inventors: Seok Hyun Yun, Yue Wu, Nicola Martino, Sheldon J J. Kwok
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Publication number: 20230272373Abstract: Methods and compositions of single tube preparation of sequencing libraries from a target DNA are provided. The methods include contacting the DNA with a composition comprising Cas9 endonuclease, a first and a second guide RNAs, a ligase, and sequencing adapters, subjecting the composition to thermal cycling to cleave the DNA at the sites flanking the regions of interest by the RNA guided endonuclease, and subjecting the composition to a temperature to allow ligation of the cleaved DNA fragments including the regions of interest with the sequencing adapters to generate the sequencing libraries.Type: ApplicationFiled: July 25, 2022Publication date: August 31, 2023Inventors: George M. Church, Benjamin W. Pruitt, Richard C. Terry
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Publication number: 20230272374Abstract: Provided are constructs and methods useful for the screening and identification of polynucleotide sequences of interest, in particular promoters, RNA stability modifying sequences and transcriptional modifying sequences.Type: ApplicationFiled: August 22, 2022Publication date: August 31, 2023Applicant: THE REGENTS OF THE UNIVERSITY OF CALIFORNIAInventors: Paul A. Feldstein, James E. Lincoln
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Publication number: 20230272375Abstract: Provided herein are methods of enriching mutated cell free nucleic acids for detection and diagnosis of cancer. Also provided are methods using a CRISPR-Cas system to target and deplete unwanted more abundant cell free nucleic acid sequences thereby enriching for less abundant sequences.Type: ApplicationFiled: April 10, 2023Publication date: August 31, 2023Inventors: Gordon Cann, Alex Aravanis, Arash Jamshidi, Rick Klausner, Richard Rava
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Publication number: 20230272376Abstract: The present invention relates to methods and compositions for editing a MECP2 polynucleotide, e.g., a MECP2 polynucleotide comprising a SNP associated with Rett syndrome. The invention also relates to methods and compositions for treating or preventing Rett syndrome in a subject.Type: ApplicationFiled: November 14, 2022Publication date: August 31, 2023Inventors: Matthew Jarpe, Mallikarjuna Reddy Putta, Andrew Fraley
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Publication number: 20230272377Abstract: The present invention relates to methods and compositions for editing a TMC1 polynucleotide, e.g., a TMC1 polynucleotide comprising a SNP associated with autosomal recessive non-syndromic hearing loss. The invention also relates to methods and compositions for treating or preventing autosomal recessive non-syndromic hearing loss in a subject.Type: ApplicationFiled: November 14, 2022Publication date: August 31, 2023Inventors: Matthew Jarpe, Mallikarjuna Reddy Putta, Andrew Fraley
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Publication number: 20230272378Abstract: This invention relates to compositions and methods for treating a disease or disorder associated with premature termination codon. Certain aspects of the invention relate to polynucleotides, vectors, and host cells, and uses thereof.Type: ApplicationFiled: June 7, 2021Publication date: August 31, 2023Applicant: University of RochesterInventors: John D. Lueck, Joseph J. Porter, Charles A. Thornton, Wooree Ko
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Publication number: 20230272379Abstract: Provided is a method for editing a target RNA at a target residue position of a host cell. The method comprises introducing ADAR-recruiting RNA (arRNA) or a construct encoding the arRNA into a host cell, wherein the arRNA comprises a complementary RNA sequence that hybridizes to a target RNA; the target residue is located in a three-base motif comprising a 5? nearest-neighbor residue (upstream residue) of the target residue in the target RNA, the target residue, and a 3? nearest-neighbor residue (downstream residue) of the target residue in the target RNA, wherein the three-base motif is not UAG, and the complementary RNA sequence comprises a mismatch directly opposite to the upstream residue or the downstream residue in the target RNA. Further provided are arRNA for the method, an RNA obtained by the method, a host cell comprising the RNA, and use of the method in the treatment of a disease.Type: ApplicationFiled: July 6, 2021Publication date: August 31, 2023Inventors: Pengfei YUAN, Zexuan YI, Nengyin LIU
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Publication number: 20230272380Abstract: A functional engineered guide RNA sequence is provided including a spacer sequence and a scaffold sequence, wherein the scaffold sequence includes a primer binding site for reverse transcription.Type: ApplicationFiled: May 8, 2023Publication date: August 31, 2023Inventors: Reza Kalhor, Prashant G. Mali, George M. Church
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Publication number: 20230272381Abstract: An object is to provide a technique that can enhance the binding properties of a single-stranded polynucleotide comprising a palindromic structure comprising an acyclic polynucleotide structural unit to target polynucleotides, such as miRNA, while suppressing self-duplex formation of the single-stranded polynucleotide. This object is achieved by a single-stranded polynucleotide comprising a palindromic structure comprising an acyclic polynucleotide structural unit, wherein adenine in the palindromic structure is replaced by diaminopurine, and thymine at a position complementary to the adenine is replaced by a thiouracil derivative.Type: ApplicationFiled: January 29, 2021Publication date: August 31, 2023Applicant: National University Corporation Tokai National Higher Education and Research SystemInventors: Hiroyuki ASANUMA, Yukiko KAMIYA, Fuminori SATO, Keiji MURAYAMA, Junya YOKOYAMA
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Publication number: 20230272382Abstract: The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the complement component C3 gene and methods of using such iRNA agents for treating or preventing C3-associated ocular diseases or C3-associated neurodegenerative diseases.Type: ApplicationFiled: September 1, 2022Publication date: August 31, 2023Inventors: Mark Keating, James D. McIninch
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Publication number: 20230272383Abstract: The present invention relates to methods and compositions for editing a GJB2 polynucleotide, e.g., a GJB2 polynucleotide comprising a SNP associated with autosomal recessive non-syndromic hearing loss. The invention also relates to methods and compositions for treating or preventing autosomal recessive non-syndromic hearing loss in a subject.Type: ApplicationFiled: November 14, 2022Publication date: August 31, 2023Inventors: Matthew Jarpe, Mallikarjuna Reddy Putta, Andrew Fraley
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Publication number: 20230272384Abstract: The present invention relates to methods and compositions for editing an RS1 polynucleotide, e.g., an RS1 polynucleotide comprising a SNP associated with X-linked retinoschisis (XLRS). The invention also relates to methods and compositions for treating or preventing XLRS in a subject.Type: ApplicationFiled: November 14, 2022Publication date: August 31, 2023Inventors: Matthew Jarpe, Mallikarjuna Reddy Putta, Andrew Fraley
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Publication number: 20230272385Abstract: The present invention relates to nucleic acids for inhibiting expression of a target gene in a cell, comprising at least one duplex region that comprises at least a portion of a first strand and at least a portion of a second strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene to be inhibited. The first strand of the nucleic acid has a terminal 5?(E)-vinylphosphonate nucleotide that is linked to the second nucleotide in the first strand by a phosphodiester linkage.Type: ApplicationFiled: November 23, 2022Publication date: August 31, 2023Inventors: Adrien Weingärtner, Lucas Bethge
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Publication number: 20230272386Abstract: A myoregulin inhibitor such as an antisense oligonucleotide against myoregulin or an anti-myoregulin antibody is used as an active ingredient of a prophylactic or therapeutic agent for a muscle disease such as muscular dystrophy, inclusion body myositis, amyotrophic lateral sclerosis, disused muscular atrophy, and sarcopenia.Type: ApplicationFiled: July 16, 2021Publication date: August 31, 2023Applicant: MITSUBISHI TANABE PHARMA CORPORATIONInventors: Kiyoshi ISHIKAWA, Shumpei MURATA
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Publication number: 20230272387Abstract: Provided herein are methods for increasing levels of SCN2A protein in a cell, comprising contacting the cell with an antisense oligonucleotide that enhances splicing at a splice site of a retained intron in an intron-retaining SCN2A mRNA or pre-mRNA, wherein the retained intron is selected from among intron 1, 2, 3, 4, 5, 11, 13, 17 and 24 and wherein the antisense oligonucleotide comprises a sequence of nucleobases that is complementary to a target region in the SCN2A mRNA or pre-mRNA. Also provided are antisense oligonucleotides for use in such methods. Also provided are methods for treating disorders associated with a heterozygous loss-of-function mutation in SCN2A, comprising administering to the subject such antisense oligonucleotides.Type: ApplicationFiled: July 22, 2021Publication date: August 31, 2023Inventor: Steven Petrou
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Publication number: 20230272388Abstract: Provided a novel miR-96-5p inhibitor that is more effective than an antisense oligonucleotide against miR-96-5p, and a novel pharmaceutical composition comprising the same. The miR-96-5p inhibitor comprises a peptide nucleic acid moiety comprising a nucleotide sequence complementary to miR-96-5p.Type: ApplicationFiled: July 29, 2021Publication date: August 31, 2023Applicant: TEIKYO UNIVERSITYInventors: Toshio NAKAKI, Koji AOYAMA, Chisato KINOSHITA, Nobuko MATSUMURA, Kazue UTSUMI, Toru SUGIYAMA, Shun-suke MORIYA
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Publication number: 20230272389Abstract: The present invention relates to miRNA interference technology. More specifically the invention relates to circular miRNA sponges that carry a plurality of binding sites directed to at least two types of miRNA and separated by random, non-identical spacers, allowing for the inhibition of functional classes of m1RNAs. Preferably, the binding sites are bulged binding sites wherein each bulge is created by a one base deletion and two base mismatch at positions 9-11 nt from the 3? end of each binding site. Preferably, each spacer is 6 to 24 nucleotides in length. Preferably, the binding sites are against miR-132 and miR-212, miR-17-5p and miR-18a-5p, or miR-20b-5p and miR-106a-5p. Construction vectors and uses of said miRNA sponges for the treatment of diseases, such as cardiomyopathy and cancer, are also disclosed.Type: ApplicationFiled: July 2, 2021Publication date: August 31, 2023Applicant: National University of SingaporeInventors: Annadoray Lavenniah, Roger Foo, Matthew Ackers-Johnson
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Publication number: 20230272390Abstract: Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed. Specifically, the disclosure provides methods of increasing the level of human fetal hemoglobin and/or gamma-globin in a cell or subject, the method comprising administering at least one zinc finger protein 410 (ZNF410) inhibitor to the cell or subject, wherein the subject has a beta-chain hemoglobinopathy, thalassemia, and/or sickle cell disease.Type: ApplicationFiled: July 29, 2021Publication date: August 31, 2023Inventors: Gerd Blobel, Xianjiang Lan, Junwei Shi
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Publication number: 20230272391Abstract: Disclosed herein are compositions and methods for recruiting and using ADAR. In some aspects, compositions and methods for targeting ADAR activity to a desired region, sequence, or nucleotide are disclosed. In some aspects, compositions and methods for increasing the efficiency of directed ADAR editing are disclosed.Type: ApplicationFiled: July 30, 2021Publication date: August 31, 2023Applicant: ADARx Pharmaceutical, Inc.Inventors: Zhen Li, Rui Zhu, Jean da Silva Correia, Kimberly Fultz, Sean Studer, Sam Lear