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WO/2024/102942A1 |
Provided are methods for determining that a candidate polypeptide in a library of polypeptides has editing activity comprising expressing in a plurality of cells: a library of first expression cassettes each comprising a nucleic acid enc...
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WO/2024/101385A1 |
Pluripotent stem cells with improved differentiation efficiency are produced while suppressing spontaneous differentiation toward all three germ layers. Pluripotent stem cells are subjected to suspension culture in a liquid medium contai...
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WO/2024/044766A3 |
Disclosed are methods and compositions for inhibiting endocytosis and enhancing function of hematopoietic stem cells. Certain aspects are directed to increasing MYCT1 activity or expression to improve hematopoietic stem cell self-renewal...
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WO/2023/069967A8 |
Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional CDKL5 (hCDKLK5). Also provided are a production system useful for producing th...
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WO/2024/101945A1 |
The present invention relates to a nanoparticle, a nanoparticle complex comprising anti-5'-tRH-GlyGCC bound thereto, and a pharmaceutical composition for preventing or treating cancer comprising same. The present invention may provide an...
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WO/2024/102703A2 |
The disclosure provides for recombinantly modified Zika-based vectors that can be used in gene therapy applications.
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WO/2024/099265A1 |
An engineered immune cell is provided, and in particular, an immune cell modified to co-express a chimeric antigen receptor (CAR) and interleukin-15 (IL-15), a pharmaceutical composition thereof, a use method therefor, and related use th...
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WO/2024/102995A1 |
Disclosed herein, are compositions and methods using Chlamydia Muridarum organisms in the treatment and prevention of human Chlamydia trachomatis infections in a subject. Also, disclosed herein are compositions and methods for eliciting ...
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WO/2024/099163A1 |
Disclosed is use of a Zea mays amino acid transporter and a coding gene thereof in plant disease resistance. Specifically disclosed are a protein having the amino acid sequence of SEQ ID No. 3 and a coding gene thereof. The present inven...
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WO/2024/098353A1 |
Provided is a recombinant Saccharomyces cerevisiae for producing cannabigerolic acid. The recombinant Saccharomyces cerevisiae is prepared from basic Saccharomyces cerevisiae, wherein in the basic Saccharomyces cerevisiae, the following ...
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WO/2024/050356A3 |
The present disclosure is directed to antibodies and antigen binding fragments thereof, or combinations of antibodies and antigen binding fragments thereof, having binding specificity for the S protein of coronaviruses (CoV-S), such as t...
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WO/2024/103051A2 |
Disclosed are exosomes comprising an extracellular HIV gpl20 binding motif. Disclosed are exosomes comprising an extracellular CD4 binding motif. Disclosed are methods of making an engineered mesenchymal stem cell (MSC)-derived exosome c...
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WO/2024/064858A3 |
Provided are oligomeric compounds, methods, and pharmaceutical compositions for reducing the amount or activity of methyl CpG binding protein 2 (MECP2) RNA in a cell or animal, and in certain instances reducing the amount of MECP2 protei...
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WO/2024/059667A3 |
The disclosure is directed in part to variant capsid polypeptides that can be used to deliver pay loads.
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WO/2024/098939A1 |
Provided is a bifunctional fusion protein, comprising a first domain specifically recognizing interleukin-6 receptor (IL-6R), and a second domain specifically binding to cell surface protein CD47. Also provided is a method of using said ...
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WO/2024/101710A1 |
The present invention relates to a pharmaceutical composition for prevention or treatment of glioma, comprising a multiple-epitope peptide, and more specifically to a technique using a multiple-epitope peptide for the use of prevention, ...
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WO/2023/212744A3 |
Provided herein are methods for diagnosing ALS, or determining risk of developing ALS. The methods include detection of a fusion as described herein. The methods can include detecting genomic fusions, fused transcripts, or fusion protein...
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WO/2024/098152A1 |
This disclosure relates to catalytic nucleic acids, catalytic nucleic acid probes, biosensors, lateral flow devices, and kits for detecting the presence or absence of eosinophil peroxidase. Also provided are methods for detecting the pre...
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WO/2024/102867A1 |
Agents, compositions, and methods of minimizing dysregulation of Staufen1-associated RNA metabolism are disclosed and described.
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WO/2024/101303A1 |
An evaluation method of an anticancer agent that includes the following steps in this order: a step of culturing a cell structure containing stromal cells, in which a gene encoding a viable cell marker protein has been knocked out, and c...
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WO/2024/102361A1 |
A pharmaceutical combination includes a beta-lactam antibiotic alone or in combination with a beta-lactamase inhibitor useful for treating multiple myeloma, monoclonal gammopathy of undetermined significance, and smoldering multiple myel...
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WO/2023/076272A9 |
In certain embodiments, provided are methods for increasing the efficiency of dicot and gymnosperm transformation using BBM with no or minimal plant phenotype. Vector designs for achieving these results are also provided, as are methods ...
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WO/2024/102996A1 |
Systems, methods, and compositions can be used to generate a therapeutic cell. The system can be an fully closed, automated system. The system, methods and compositions can intake cells of a subject and modify the cells to generate thera...
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WO/2024/064683A3 |
Compositions comprising an RNA molecule comprising a guide sequence portion having 17- 50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-34194 and methods and uses thereof.
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WO/2024/081673A3 |
The present disclosure provides, among other things, systems, engineered cell lines and methods of manufacturing recombinant viral vectors and products. Engineered cell lines comprise one, two, three, or more stably integrated genomic co...
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WO/2024/102965A1 |
Disclosed herein are designer extracellular vesicles (EVs) that target muscle cells. For example, in some embodiments, the EVs are decorated NHERF1, NHERF2, a fusion protein containing an E8 fragment of laminin and an exosomal or lysosom...
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WO/2024/102667A2 |
The disclosure relates to gene editing systems comprising serine recombinases and methods of using such serine recombinases for integration of nucleic acid sequences.
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WO/2024/098116A1 |
The present invention relates to CRISPR/Cas-based biosensing materials, assays and methods. In particular, the technology relates to ultrasensitive CRISPR/Cas-based detection methods for nucleic acid assays using special molecular constr...
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WO/2024/102556A1 |
The present disclosure is generally related to genetically modified filamentous fungal strains (cells) and their use in the production of proteins of interest. Certain embodiments provide, inter alia, methods and compositions for the des...
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WO/2024/099320A1 |
A hybrid protein containing a CFH functional unit and a DAF functional unit, and a mutant thereof, an encoding nucleic acid thereof, and a method and the use thereof for treating diseases or conditions related to a complement system; and...
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WO/2024/044750A3 |
The present disclosure features allogeneic modified cells (e.g., T- or NK-cells) having increased persistence, increased resistance to immune rejection, or decreased risk of eliciting a host-versus-graft reaction, or a combination thereo...
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WO/2024/050547A3 |
Adeno-associated viruses (AAV) provide a safe means of therapeutic gene delivery; however, a significant technical obstacle limits an AAV vector's utility: its small payload capacity. The large size of certain genes, in addition to a pro...
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WO/2024/101433A1 |
A glycosylated neuropeptide derivative comprising a neuropeptide sequence, a membrane-penetration-promoting sequence, an endosomal-escape-promoting sequence, and a sugar chain.
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WO/2024/101294A1 |
[Problem] To provide a method for obtaining a sequence for a nucleic acid including an artificial nucleic acid, more specifically a method for amplifying a sequence for a nucleic acid including a non-cyclic artificial nucleic acid to amp...
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WO/2024/101962A1 |
The present invention relates to cells that have been genetically engineered to express a peptide or fragment thereof capable of inhibiting the transforming growth factor-β (TGF-β) signaling pathway, in conjunction with a chimeric anti...
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WO/2024/102799A1 |
The present disclosure relates, generally, to compositions and methods for producing, purifying, and using circular RNA.
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WO/2024/102677A1 |
Provided herein are circular RNA constructs comprising an IRES, and at least one expression sequence encoding binding molecule, compositions thereof, and methods of treatment, including for cancer and autoimmune disease. In particular, c...
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WO/2024/102436A1 |
Provided herein is an engineered controllable membrane targeting system based on triggered exposure of a cryptic acylation signal, a calcium-regulated protease that can be used to expose this cryptic acylation signal, a means of coupling...
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WO/2024/076526A3 |
The present invention is directed to an agricultural mixture comprising 1-amino-1- cyclopropanecarboxylic acid, a hydrate thereof, a polymorph thereof or a salt thereof and prohydrojasmon. The present invention is further directed to a m...
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WO/2024/102761A1 |
Provided herein are methods of detecting tumor nucleic acids in a biological sample of a subject.
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WO/2024/100557A1 |
Provided are glycoprotein NMB (GPNMB)-binding chimeric antigen receptors (CARs). In some embodiments, a CAR of the present disclosure comprises an extracellular GPNMB-binding domain (e.g., a single chain antibody, such as an scFv), a tra...
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WO/2024/102739A2 |
A method of making a viral particle packaging and producer ceil line is provided. The cell line is based on an adeno-associated virus (AAV) vector, the cell line is caused to expresses, in association with the viral particle, a desired t...
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WO/2024/102947A1 |
The present disclosure provides methods for transcriptionally modulating genomic target sites in a cell comprising contacting a cell with: a variant type V CRISPR/Cas effector polypeptide fused to one or more heterologous polypeptides, w...
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WO/2024/098369A1 |
Disclosed herein are an isolated microRNA molecule, a Parkinson's disease in-vitro diagnostic kit based on a DNA hexahedron, and use thereof. The microRNA molecule miR26690 provided by the present application is highly expressed in an ex...
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WO/2024/101382A1 |
The present disclosure addresses the problem of providing a novel composition to be used in immune checkpoint regulation. One aspect of the present disclosure provides a composition which is to be used in immune checkpoint regulation and...
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WO/2024/081352A3 |
The present disclosure provides third-generation self-inactivating (SIN) lentiviral vectors comprising a nucleic acid sequence encoding MyoVIIa isoform 1 for treatment of Usher IB syndrome in a subject in need thereof. In some embodiment...
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WO/2022/117731A9 |
The present invention relates to a method of generating a nanoparticle comprising (c) contacting an antibody with a composition comprising a first conjugate (A), the first conjugate comprising a positively charged polypeptide conjugated ...
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WO/2024/102811A2 |
Provided herein are compositions and methods for treating or preventing dilated cardiomyopathy (DCM) in a subject in need thereof, e.g., through correcting one or more point mutations at the RBM20 locus using CRISPR-associated base editing.
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WO/2024/036186A3 |
In some embodiments, described herein are antisense oligonucleotides that target a C90rf72 sequence comprising a two-base pair deletion and G4C2 repeat expansions; and methods of using same to inhibit C90rf72 expression. In some embodime...
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WO/2024/103049A1 |
Disclosed are exosomes comprising an extracellular CD33 binding motif fused to a transmembrane protein. Disclosed are methods of making a cell derived exosome comprising: transfecting a plasmid into cells, wherein the plasmid comprises a...
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