Abstract: Engineered immune cells, optionally natural killer (NK) cells, comprising an inactivating mutation for an endogenous A-Disintegrin-And-Metalloproteinase 17 (ADAM 17) gene. ADAM17-deficient immune cells produced from stem cells, optionally, induced pluripotent stem cells (iPSCs), having enhanced antibody-dependent cellular cytotoxicity (ADCC). Pharmaceutical compositions comprising said engineered immune cells. Methods for making the engineered immune cell and pharmaceutical compositions, and methods of use are provided.
Abstract: Use of a composition comprising a combination of a urolithin, a NAD precursor, preferably Nicotinamide Riboside and Vitamin B12 for increasing stem cell function in a population of haematopoietic stem and/or progenitor cells (HSPCs).
Type:
Application
Filed:
September 28, 2021
Publication date:
January 11, 2024
Inventors:
NICOLA VANNINI, MUKUL GIROTRA, GEORGE COUKOS
Abstract: The present invention relates to a method for inducing differentiation, into chondrocytes, of cord blood mononuclear cell-derived induced pluripotent stem cells. In a case where a chondrogenic pellet produced by the method of the present invention is transplanted into a cartilage damage area in vivo, regeneration of cartilage can be effectively exhibited by differentiated chondrocytes. In such a case, an effective cartilage regeneration capacity can be exhibited as compared with a case where chondrocytes produced by differentiation induction with the addition of a recombinant growth factor are transplanted. Thus, the present invention can be usefully used for tissue engineering therapies.
Abstract: The present invention is directed toward edible hollow fibers and cartridges and bioreactors comprising the hollow fibers of the present invention, as well as, methods of production of structured clean meat products produced with the hollow fibers, cartridges and bioreactors of the present invention and the structured clean meat products produced by said methods. The macroscopic structure of structured clean meat grown on edible hollow fibers will result in a unique final structure. This final structure will contain a finite amount of fibers per unit area; with meat on the outside of the fibers.
Type:
Application
Filed:
August 19, 2021
Publication date:
January 11, 2024
Inventors:
Jean-Louis Weissenbach, Ryan Sylvia, Almut Von der Brelie, Melanie Brandl, Michaela Fesenfeld
Abstract: The invention relates inter alia to a method for differentiating a muscle progenitor cell, comprising the step of: —culturing a muscle progenitor cell in a serum-free medium for differentiating a muscle progenitor cell, wherein said serum-free medium comprises —at least one differentiation inducer selected from the group consisting of a lysophosphatidic acid receptor 1 (LPAR1) agonist, a lysophosphatidic acid receptor 3 (LPAR3) agonist, an oxytocin receptor (OXTR) agonist, a glucagon receptor (GCGR) agonist, a lactate and a Notch signaling pathway inhibitor.
Type:
Application
Filed:
November 25, 2021
Publication date:
January 11, 2024
Inventors:
Helder CRUZ, Joshua Edwin FLACK, Carolina FURQUIM, Iva KLEVERNIC, Lea MELZENER, Tobias MEßMER, Mark POST, Anon VAN ESSEN
Abstract: The invention relates to a dissolvable gelatin-based microcarrier generated through droplet microfluidics. Also disclosed herein is a method of manufacturing said microcarrier and its use in the processes of cell culture and cell expansion of cells, such as mesenchymal stromal cells (MSCs).
Abstract: A method for culturing mesenchymal stem cells is disclosed. The method includes steps of: primary culturing mesenchymal stem cells in a medium containing umbilical cord blood serum; and subculturing the mesenchymal stem cells in a medium containing fetal bovine serum. The method for culturing mesenchymal stem cells may maintain stemness by reducing the differentiation and aging of stem cells, and in particular, may achieve a remarkably high proliferation rate.
Type:
Application
Filed:
November 9, 2021
Publication date:
January 11, 2024
Applicants:
ENCELL CO., LTD., SAMSUNG LIFE PUBLIC WELFARE FOUNDATION
Inventors:
Hong Bae JEON, Sang Eon PARK, Jong Wook CHANG
Abstract: The present invention relates to an in vitro liver disease model using triple co-culture and a preparation method thereof, and the method of preparing the in vitro liver disease model includes directly co-culturing hepatocytes and hepatic stellate cells, and simultaneously indirectly co-culturing Kupffer cells by being separated from the hepatocytes and the hepatic stellate cells to enable a rapid and highly accurate analysis by similarly implementing the in vivo environment compared to mono-culture, and simultaneously make it easier to collect and measure specimens than typical direct co-culture.
Type:
Application
Filed:
November 24, 2021
Publication date:
January 11, 2024
Applicant:
FUTURE MEDICINE CO., LTD.
Inventors:
Seong Wook SEO, Yoon Pyo CHOI, Sang Yeop AHN, Da In PARK, Myeong Hoon LEE
Abstract: Provided are engineered cells, such as engineered primary cells, containing one or more modifications, such as genetic modifications, for use in allogeneic cell therapy. In some embodiments, the engineered primary cells are hypoimmunogenic cells.
Abstract: A medium for culturing and expanding nephron progenitor cells, the medium containing a GSK-3? inhibitor, a ROCK inhibitor, and at least one fibroblast growth factor selected from the group consisting of FGF9 and FGF20. Also, a method for culturing and expanding nephron progenitor cells using the medium. Also, a method for producing renal organoids, the method including a step of culturing and expanding nephron progenitor cells using the above method for culturing and expanding nephron progenitor cells, and a step of differentiating the cultured and expanded nephron progenitor cells into renal organoids.
Abstract: The present invention concerns a novel in vitro assay for determining the invasive ability of brain tumour cells, in particular brain tumour stem cells. This new assay, called 3D invasion assay, is based on the analysis and comparison of the area of neurospheres grown in vitro into a Matrigel extracellular-like matrix. The invention also concerns the use of said assay for diagnosing metastatic brain tumour in subjects, for determining its prognosis, as well as for the monitoring, for the stratification, for identifying subjects at risk of metastasis, and/or predicting the metastasis-associated risk in subjects diagnosed with brain tumour.
Type:
Application
Filed:
September 14, 2021
Publication date:
January 11, 2024
Applicant:
Institut Gustave Roussy
Inventors:
David CASTEL, Marie Anne DEBILY, Jacques GRILL, Marco BRUSHI
Abstract: Provided herein are compositions and methods to promote longevity of induced pluripotent stem cell (iPSC)-derived endothelial cells (ECs) via autophagy. In particular, provided herein are AMP-activated protein kinase (AMPK) activators, such as ginsenosides (e.g., Rg2), and use thereof to increase longevity of iPSC-ECs by stimulating mTOR-independent ULK1-mediated autophagy.
Type:
Application
Filed:
July 11, 2023
Publication date:
January 11, 2024
Inventors:
Katherine E. Hekman, Jason A. Wertheim, Congcong He
Abstract: A cell culture chamber is for the in vitro production and cultivation of cell layers and organ models with two first and second channels arranged one above the other and separated from one another by a porous membrane with two side surfaces and through which flow can pass, wherein a cell substrate is formed in each case by the side surfaces of the membrane. The cell culture chamber is characterized in that at least the inner walls of the first and the second channels consist of polybutylene terephthalate. Further, a method is for cultivating human or animal cells, and in particular liver sinusoidal endothelial cells, alone and in co-culture with hepatocytes and immune cells.
Abstract: The present invention concerns a production bacterial cell for producing lytic phage particles or lytic phage-derived delivery vehicles, said production bacterial cell stably comprising at least one phage structural genes and at least one phage DNA packaging genes, said phage structural gene(s) and phage DNA packaging gene(s) being derived from a lytic bacteriophage, wherein the expression of at least one of said phage structural genes and/or at least one of said phage DNA packaging gene(s) in said production bacterial cell is controlled by an induction mechanism.
Type:
Application
Filed:
July 7, 2023
Publication date:
January 11, 2024
Inventors:
Jesus FERNANDEZ RODRIGUEZ, Antoine DECRULLE, Aymeric LEVEAU, Ines CANADAS BLASCO, Aurelie MATHIEU, Thibault CARLIER
Abstract: Modified influenza virus neuraminidases are described herein that have stabilized NA tetramers which may improve vaccine production efficiency, thus improving the yield of vaccine viruses.
Abstract: Novel polypeptides having peroxygenase activity, and methods and uses related thereto. A method for the production of melanin or a melanin-like pigment, comprising the use of a polypeptide having pigment producing activity, wherein said polypeptide is selected from the group consisting of: (a) a polypeptide comprising an amino acid sequence having at least 50% pairwise sequence identity when aligned to at least 200 consecutive amino acid residues of Seq. no. 16 and comprising at least two of the following motifs: i) RXFWXRWXXGHQ; ii) LXXLXXCXD; iii) PRXXYH; iv) RXR[ML]ALQH; v) CXXL; vi) HXXIAXH; vii) DLXHXG; and viii) VDGXHHPV; (b) a polypeptide comprising an amino acid sequence having at least 30% pairwise sequence identity when aligned to at least 150 amino acid residues of Seq. no. 12, and comprising the motif HXXXC; and c) a fragment of the polypeptide of (a) or (b) that has peroxygenase activity.
Abstract: Provided are a cytochrome P450 enzyme mutant and an application thereof. The enzyme activity and anti-Markov oxidation selectivity of P450 derived from a wild-type strain of Bacillus megaterium undergo protein modification by means of directed evolution, thus improving enzyme activity and selectivity, and developing a series of P450 enzyme mutants that may be used for industrial production.
Type:
Application
Filed:
April 25, 2021
Publication date:
January 11, 2024
Inventors:
Hao HONG, Gage JAMES, Na ZHANG, Xuecheng JIAO, Yulei MA, Shan CAO, Yibing CHENG, Kaihua SUN
Abstract: Transgenic plants with modified flower color, or their inbred or outbred progeny, or their propagates, partial plant bodies, tissues or cells, are provided. A delphinidin-type anthocyanin and a flavone C-glycoside in which the 7-position and T-position hydroxyl groups are methylated, are caused to coexist in plant cells.
Abstract: The present invention provides engineered glycosyltransferase (GT) enzymes, polypeptides having GT activity, and polynucleotides encoding these enzymes, as well as vectors and host cells comprising these polynucleotides and polypeptides. The present invention provides engineered sucrose synthase (SuS) enzymes, polypeptides having SuS activity, and polynucleotides encoding these enzymes, as well as vectors and host cells comprising these polynucleotides and polypeptides. The present invention also provides compositions comprising the GT enzymes and methods of using the engineered GT enzymes to make products with ?-glucose linkages. The present invention further provides compositions and methods for the production of rebaudiosides (e.g., rebaudioside M, rebaudioside A, rebaudioside I, and rebaudioside D). The present invention also provides compositions comprising the SuS enzymes and methods of using them. Methods for producing GT and SuS enzymes are also provided.
Type:
Application
Filed:
August 2, 2023
Publication date:
January 11, 2024
Inventors:
Jonathan Vroom, Stephanie Sue Galanie, Jack Liang, Joyce Liu, Nikki Dellas, Melissa Ann Mayo, David Entwistle
Abstract: Disclosed are recombinant fusion proteins comprising a villin headpiece HP47 domain and a heterologous protein domain, such as, for example, a nucleic acid polymerase. Also disclosed are nucleic acids (e.g., DNA constructs) encoding the fusion protein, expression vectors and recombinant host cells for expression of the fusion protein, and methods of using the recombinant fusion proteins.
Type:
Application
Filed:
September 15, 2021
Publication date:
January 11, 2024
Inventors:
Andrew ELLINGTON, Inyup PAIK, Andre MARANHAO, Sanchita BHADRA, David WALKER, Phuoc NGO, Daniel DIAZ
Abstract: The invention relates to a GDSL lipase, genetically-engineered bacteria and an application thereof. The GDSL lipase is derived from Streptomyces diastaticus CS1801 and its amino acid sequence is as shown in SEQ ID NO.2. After construction of a genetically-engineered bacterium strain, a GDSL lipase is generated through fermentation. Through this enzyme, vitamin A and palmitic acid are converted to produce vitamin A palmitate. The content of the vitamin A palmitate obtained from the conversion is 16.35 mg/L at most. The conversion efficiency is 81.75% at most. This lipase provides a new path to synthesize vitamin A palmitate by the enzymatic method and has an important application prospect.
Type:
Application
Filed:
June 27, 2023
Publication date:
January 11, 2024
Applicants:
Suzhou Kemanduo Biotechnology Co., Ltd., Changshu Institute of Technology
Inventors:
Bin QI, Manting QI, Limei WANG, Yuhua WANG
Abstract: The invention relates, in part, to methods of preparing male organisms of a species that include engineering the male organisms in a manner that eliminates post-embryonic viability of the female offspring and female descendants of the engineered male organisms. Certain aspects of the invention include use of such prepared engineered male organisms in methods of controlling population levels of organisms of the species.
Abstract: The present invention encompasses engineered meganucleases that bind and cleave a recognition sequence within a TTR gene. The present invention also encompasses methods of using such engineered meganucleases to make genetically-modified cells. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, or nucleic acids encoding engineered meganucleases of the invention, and the use of such compositions for treatment of TTR-associated diseases, such as transthyretin amyloidosis.
Type:
Application
Filed:
August 20, 2021
Publication date:
January 11, 2024
Applicant:
Precision Biosciences, Inc.
Inventors:
Cassandra Gorsuch, Jochen Genschel, Janel Lape, Derek Jantz, James Jefferson Smith
Abstract: The present invention relates to variant CRISPR nuclease polypeptides, methods of preparing the variant CRISPR nuclease polypeptides, processes for characterizing the variant CRISPR nuclease polypeptides, compositions and cells comprising the variant CRISPR nuclease polypeptides, and methods of using the variant CRISPR nuclease polypeptides. The invention further relates to complexes comprising a variant CRISPR nuclease polypeptide, methods of producing the complexes, processes for characterizing the complexes, cells comprising the complexes, and methods of using the complexes.
Type:
Application
Filed:
January 7, 2022
Publication date:
January 11, 2024
Inventors:
Shaorong CHONG, Brendan Jay HILBERT, Victor Mikhailovich GUZOV, Austin McCarty JONES
Abstract: The disclosure provides Cas12f polypeptides, fusion proteins comprising such Cas12f polypeptides, CRISPR-Cas12f systems comprising such Cas12f polypeptides or fusion proteins, and methods of using the same.
Type:
Application
Filed:
June 8, 2023
Publication date:
January 11, 2024
Inventors:
Xiangfeng Kong, Lecong Wang, Xuqiang Kong
Abstract: The invention provides methods of inactivating biological activity of RNA based therapeutics, such as vaccines, through administration of compositions possessing or stimulating RNAse activity alone or together with lipid based formulations capable of delivering RNAse or RNAse stimulating activity into said RNAse based therapeutic.
Abstract: Provided herein are compositions and methods of using prime editing systems comprising prime editors and prime editing guide RNAs for treatment of genetic disorders.
Type:
Application
Filed:
August 14, 2023
Publication date:
January 11, 2024
Inventors:
Jennifer Gori, David Waterman, Jack Heath, Andrew V. Anzalone
Abstract: Modified PH20 hyaluronidase polypeptides, including modified polypeptides that exhibit increased stability and/or increased activity, are provided. Also provided are compositions and formulations and uses thereof.
Type:
Application
Filed:
June 20, 2023
Publication date:
January 11, 2024
Inventors:
Ge WEI, H. Michael Shepard, Qiping Zhao, Robert James Connor
Abstract: Provided herein are modified dipeptide cleavases for removing amino acid(s) from peptides, polypeptides, and proteins. Also provided are methods of using the modified dipeptide cleavases for treating polypeptides, and kits comprising the modified dipeptide cleavase. In some embodiments, the methods and the kits also include other components for macromolecule sequencing and/or analysis.
Type:
Application
Filed:
March 19, 2021
Publication date:
January 11, 2024
Applicant:
Encodia, Inc.
Inventors:
Kevin L. GUNDERSON, Robert C. JAMES, Lei SHI, Stephen VERESPY, III, Kevin DESAI
Abstract: Disclosed herein include methods, compositions, and kits suitable for use in winner-take-all neural network computation in mammalian cells. In some embodiments, de novo designed protein heterodimers and engineered viral proteases are combined to implement a synthetic protein circuit that performs winner-take-all neural network computation. The synthetic protein circuit can include modules that compute weighted sums of input protein concentrations through reversible binding interactions, and allow for self-activation and mutual inhibition of protein components using irreversible proteolytic cleavage reactions.
Abstract: The present invention relates to means and methods for protecting proteins and protein-type compounds in industrial and other applications. In particular, the invention provides a composition comprising at least one protein or protein-type compound immobilized at the surface of a solid carrier embedded in a protective material. Further, the present invention relates to methods for producing such a composition and to the use thereof in, for example, therapeutic applications. In particular, the system may be used to immobilize and protect enzymes on the surface of a carrier to generate a biocatalytical composition with increased resistance to various types of stresses.
Type:
Application
Filed:
July 12, 2023
Publication date:
January 11, 2024
Inventors:
Patrick Shahgaldian, Maria Rita Correro-Shahgaldian, Alessandro Cumbo, Philippe Francois-Xavier Corvini
Abstract: The present disclosure describes improved methods for use in purifying biological products made by host cells. In some embodiments, the improved methods comprise one or more steps of lysing host cells, such as with a detergent, to release the biological product, precipitating host cell DNA, such as with domiphen bromide, and then inhibiting precipitation of residual host cell DNA in a supernatant containing the biological product by adding a salt to a sufficient final concentration. In some embodiments, the biological product is a vaccine, or a viral vector for gene therapy, such as an AAV vector or a lentiviral vector.
Type:
Application
Filed:
December 17, 2021
Publication date:
January 11, 2024
Inventors:
Neha KALLA, William KISH, John LIGHTHOLDER, Zhuo LIU, Eric VORST, Tamara ZEKOVIC
Abstract: Provided is a nucleic acid purification method capable of efficiently purifying a nucleic acid. The nucleic acid purification method including the steps of: mixing a nucleic acid with an antifoaming agent and a coprecipitation agent; and purifying the nucleic acid, in which the antifoaming agent is at least one of a nonionic surfactant and a silicone antifoaming agent.
Type:
Application
Filed:
September 3, 2021
Publication date:
January 11, 2024
Applicants:
SEKISUI MEDICAL CO., LTD., SEKISUI CHEMICAL CO., LTD.
Abstract: A method may be implemented for in-tip flow-through magnetic bead processing. A biological solution may include a plurality of magnetic beads suspended therein. The biological solution may be introduced to a tube via an opening in a tip portion of the tube. The tip portion of the tube may include a magnetizable material arranged in a flow path of the biological solution. The magnetizable material may include a ferromagnetic matrix or a wire within the tip portion. A magnetic field may be applied proximate to the tip portion of the tube using an electromagnetic coil. The electromagnetic coil may be wound around the tip portion. The biological solution may be removed from the tube, for example, via the opening in the tip portion. The plurality of magnetic beads may be captured within the magnetizable material in the tip portion using the magnetic field.
Abstract: Provided herein is an antigen display library for detecting antibodies produced by an individual; and methods of using the antigen display library to generate an antibody signature, the method comprising contacting a biological sample containing antibodies from an individual with the antigen display library, isolating phage clones displaying antigenic epitopes recognized by antibody in the sample, and identifying the antigenic epitopes that were recognized by antibody in the sample. Also provided are kits for generating an antibody signature comprising the antigen display library, a substrate for isolating phage clones bound by antibody, and may further comprise reagents useful for generating the antibody signature.
Abstract: A Saccharomyces cerevisiae antibody display system that simultaneously secrets and displays antibody fragments from a very large size synthetic nave human antibody library for therapeutic antibody lead discovery and engineering is described. A bait anchor complexed with a monovalent antibody fragment is tethered on the surface of the S. cerevisiae host cell, wherein the fragment can be assayed for antigen binding, while the full bivalent antibody is simultaneously secreted from the host cell. Methods of using the system for identifying antibodies from the library that bind specifically to an antigen of interest are also provided. Polypeptides, polynucleotides and host cells used for making the antibody display system are also provided along with methods of use thereof.
Abstract: Provided herein are methods of treating or preventing herpesvirus infection comprising modulating interactions between herpesvirus surface proteins and plasma membrane-expressed host cell proteins, as well as methods of identifying modulators of such interactions.
Abstract: The invention provides methods for assessing the global levels of a plurality of different chromatin modifications in parallel in a plurality of samples. The methods disclosed herein also relate to assessing the levels of a plurality of different chromatin modifications, in a plurality of locations of interest within genome, in a plurality of samples. The methods are highly multiplexed, quantitative and involve chromatin immunoprecipitation and sequencing technology.
Abstract: Methods and compositions for multiplexed protein-protein interaction profiling (e.g., immunoprofiling), based on nucleic acid tagging of polypeptides (e.g., by RNA display) are described. In some embodiments the described compositions and methods utilize a library of prey polypeptide targets linked to prey RNAs encoding them, and a population of bait polypeptides, e.g., a mixture of antibodies, that bind to one or more of the prey polypeptide targets and are used to isolate and identify the bound prey polypeptide targets by amplification of their associated prey RNAs and sequencing of the corresponding cDNAs. In other embodiments the prey polypeptide targets are linked to DNA Bar Codes, which serve as unique identifiers of the tagged polypeptide.
Abstract: Disclosed herein are compositions, kits, and methods for amplifying a sequencing assay region of a target nucleic acid from a nucleic acid sample from any source, while simultaneously adding a plurality of barcode sequences during the amplification process, to create a library of amplified amplicons which is then sequenced, with the barcode sequences enabling identification of the nucleic acid sample from which the amplicon derives. The compositions and methods can be used, for example, to create amplicons containing combinatorial barcodes for the purposes of rapidly sequencing many nucleic acid samples for the presence of viral or mutant nucleic acids.
Abstract: Methods, systems and related compositions are provided to perform single-cell marking of a nucleic acid and/or protein in a sample based on in-cell or in-organelle barcoding of nucleic acid and/or protein complexes of the cell or organelle; the methods and systems herein described are configured to provide in-cell or in-organelle single-cell marked nucleic acid and/or protein complexes comprising a single-cell, cell-specific, or a single-cell organelle-specific marker.
Type:
Application
Filed:
February 23, 2023
Publication date:
January 11, 2024
Inventors:
Rustem F. ISMAGILOV, Matthew S. CURTIS, Mary ARRASTIA, David A. SELCK, Mitchell GUTTMAN
Abstract: The present disclosure provides compositions comprising nucleic acid double-stranded splint adaptors, including kits, and methods that employ the double-stranded splint adaptors, e.g., PCR-free workflows. The double-stranded splint adaptors (200) can be used in a one-pot, multi-enzyme reaction to introduce one or more new adaptor sequences into a library molecule. The double-stranded splint adaptor (200) comprises a first splint strand (long splint strand (300)) and a second splint strand (short splint strand (400)), where the first and second splint strands are hybridized together to form the double-stranded splint adaptor (200) having a double-stranded region and two flanking single-stranded regions. The second splint strand (400) carries the new adaptor sequence(s) to be introduced, such as for example a universal binding sequence and/or an index sequence.
Abstract: Polynucleotides are engineered to hybridize to one another to form a functional guide nucleic acid having an editing template. The hybridized guide nucleic acids may be used with template-based gene editors to write an edit into a target genomic location using the editing template as a template for the edit.
Abstract: This invention relates to synthetic CRISPR spacer polynucleotides and compositions comprising the same such as synthetic spacer-repeat sequences and synthetic CRISPR arrays, wherein the synthetic CRISPR spacer polynucleotides, when translated according to amino acid single-letter code convention, spell a text. Further provided in this invention are methods of using the same for labeling and/or detecting bacteria of interest.
Abstract: Provided herein are compositions and methods to assess the genomic landscape of fixed cells using light activated oligonucleotides that can be directed to the nucleus, mitochondria, or cytoplasm of fixed cells and that, upon activation, can be extended for in situ copying of nuclear single-stranded DNA (i.e., open chromatin), open mitochondrial DNA, and/or cytoplasmic RNA into barcoded complementary DNA. These methods also provide for gene specific 3D chromatin structural niche analysis.
Type:
Application
Filed:
June 15, 2023
Publication date:
January 11, 2024
Applicants:
Agilent Technologies, Inc., The Trustees of the University of Pennsylvania
Inventors:
James EBERWINE, Jae-Hee LEE, Jifen LI, Stephen FISHER, Youtao LU, Junhyong KIM, Jai-Yoon SUL, Jinchun WANG, Mimi HEALY
Abstract: Disclosed are methods and compositions for promoting trans-splicing. In some embodiments, the composition comprises an engineered small nuclear RNA that promotes trans-splicing of a target RNA molecule. The composition may further comprise an RNA donor molecule.
Abstract: The disclosure relates to compositions and methods for treating a disease or condition associated with a TDP-pathology or a decline in TDP-43 functionality in neuronal cells in a subject, and for identifying candidate agents to suppress or prevent inclusion of an abortive or altered STMN2 RNA sequence.
Type:
Application
Filed:
March 25, 2021
Publication date:
January 11, 2024
Inventors:
Kevin C. Eggan, Joseph Robert Klim, Robert H. Brown, Jr., Jonathan K. Watts
Abstract: Disclosed herein are compositions, pharmaceutical compositions, and methods of use comprising an engineered polynucleotide that can be used to hybridize with a target RNA which may contain a nucleotide mismatch. Compositions and methods disclosed herein can be used to edit RNA to ameliorate or treat diseases or conditions in a subject.
Abstract: Oligonucleotides comprising modifications at the 2? and/or 3? positions(s) along with methods of making and use against Alzheimer disease and other tauopathies are disclosed.
Type:
Application
Filed:
September 26, 2022
Publication date:
January 11, 2024
Inventors:
Andreas EBNETH, Constantin VAN OUTRYVE D'YDEWALLE, Sergei GRYAZNOV, Saúl MARTINEZ MONTERO, Leonid BEIGELMAN, Vivek Kumar RAJWANSHI