Abstract: Provided herein are compositions of NK-92® cells that express a CD19 CAR, CD16 and IL2, and the method of using these cells to and treat cancer in a patient.

Abstract: The present disclosure provides a heterodimeric, conditionally active chimeric antigen receptor (CAR), and a nucleic acid comprising a nucleotide sequence encoding the CAR. The present disclosure provides cells genetically modified to produce the CAR. A CAR of the present disclosure can be used in various methods, which are also provided.

Abstract: The present invention relates to an engineered immune cell defective for Suv39h1. Preferably, said engineered immune cell further comprises a genetically engineered antigen receptor that specifically hinds a target antigen. The present invention also relates to a method for obtaining a genetically engineered immune cell comprising a step consisting in inhibiting the expression and/or activity of Suv39h1 in the immune cell; and further optionally comprising a step consisting in introducing in the said immune cell a genetically engineered antigen receptor that specifically binds to a target antigen. The invention also encompasses said engineered immune cell for their use in adoptive therapy, notably for the treatment of cancer.

Abstract: Methods for treating a human suffering from osteoarthritis are provided. Aspects of the methods include intra-articularly administering to the human a dosage comprising a nucleic acid coding sequence for a human interleukin-1 receptor antagonist (IL-1Ra) to treat the human suffering from osteoarthritis. Also provided are compositions for use in practicing the methods.

Abstract: The present invention provides gene therapies for the treatment of Friedreich's ataxia. Specifically, the present invention provides a nucleic acid, cloning vector and transfer vector for the production of an adeno-associated virus (AAV) vector. The nucleic acid comprises (i) a nucleic acid sequence encoding frataxin, (ii) a phospho-glycerate-kinase (PGK) promoter, and (iii) a woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). The present invention also provides a pharmaceutical composition which comprises the AAV vector or nucleic acid. Also, the AAV vector, nucleic acid or pharmaceutical composition can be used as a medicament, specifically as a medicament for the treatment of Friedreich's ataxia.

Abstract: Provided are a preparation method and system for a recombinant adeno-associated virus (rAAV) and a recombinant bacmid. The method comprises: first reconstructing a recombinant bacmid containing a recombinant baculovirus genome that produces essential functional elements for an rAAV, at least one of the essential functional elements being inserted into the N-terminal or C-terminal of a locus of an essential gene of the recombinant baculovirus genome; and then transfecting the obtained recombinant bacmid containing the recombinant baculovirus genome that produces the rAAV into a host cell line for culturing to prepare an rAAV. Compared with recombinant baculoviruses obtained by conventional Tn7 recombinant preparations of recombinant bacmid, the recombinant baculovirus obtained by inserting a core element containing Cap, Rep and ITR into two sides of a baculovirus essential gene has a more stable rAAV serial passage production level in a cell and has a higher rAAV yield.

Abstract: Provided herein are methods of generating antigen-specific T cells for therapeutic administration to a human patient having or suspected of having a pathogen or cancer, utilizing soluble IL-15/IL-15R? complexes ex vivo, in cell culture during ex vivo sensitizing of T cells to the antigen or during ex vivo culturing of antigen-specific T cells. Also disclosed are antigen-specific T cells generated by such methods, and methods of treating a human patient using such antigen-specific T cells. Cell culture systems comprising human T cells, antigen-presenting cells, and soluble IL-15/IL-15R? complexes are also provided.

Abstract: The invention provides hybrid and recombinant phagemid vectors for expressing a transgene in a target cell transduced with the vector. A recombinant phagemid particle comprises at least one transgene expression cassette which encodes an agent which exerts a biological effect on the target cell, characterised in that the phagemid particle comprises a genome which lacks at least 50% of its bacteriophage genome. The invention extends to the use of such phagemid expression systems as a research tool, and for the delivery of transgenes in a variety of gene therapy applications, DNA and/or peptide vaccine delivery and imaging techniques. The invention extends to in vitro, in vivo or in situ methods for producing viral vectors, such as recombinant adeno-associated viruses (rAAV) or lentivirus vectors (rLV), and to genetic constructs used in such methods.

Abstract: Disclosed herein, in certain embodiments, are recombinant myxoma viruses (MYXVs) and nucleic acid constructs encoding the recombinant MYXVs. In some embodiments, the MYXVs are engineered to inactivate or attenuate an activity or expression level of an M153 protein. In some embodiments, the MYXVs are engineered to express one or more transgenes such as a tumor necrosis factor (TNF), interleukin-12 (IL-12), or decorin. Also disclosed herein, in certain embodiments, are methods of using the MYXVs. Some embodiments include providing a MYXV as described herein to a subject in need thereof.

Abstract: A genome-editing complex for modifying a target polynucleotide comprising a recombinant ? helical protein linked to either one or more molecules of a genome-editing system or a plasmid encoding for one or more molecules of a genome-editing system, wherein the ? helical protein length is in the range of from 5 nm to 25 nm, and width is in the range of from 1 nm to 5 nm.

Abstract: The present disclosure provides methods and compositions for genetically modifying lymphocytes, for example T cells and/or NK cells, in shorter times than previously and/or in whole blood or a component thereof. In some embodiments a lymphodepletion filter assembly is used before or after forming a reaction mixture where lymphocytes are contacted with recombinant retroviral particles in a closed system, to genetically modify the lymphocytes.

Abstract: The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.

Abstract: Enhanced virus-like particles (eVLPs), comprising a membrane comprising a phospholipid bilayer with one or more virally-derived glycoproteins on the external side; and a cargo disposed in the core of the eVLP on the inside of the membrane, wherein the eVLP does not comprise an exogenous gag/pol protein, and methods of use thereof for delivery of the cargo to cells.

Abstract: Methods and composition for modulating a target genome and stable integration of a transgene of interest into the genome of a cell are disclosed.

Abstract: Non-human animals comprising a human or humanized IL-4 and/or IL-4R? nucleic acid sequence are provided. Non-human animals that comprise a replacement of the endogenous IL-4 gene and/or IL-4R? gene with a human IL-4 gene and/or IL-4R? gene in whole or in part, and methods for making and using the non-human animals, are described. Non-human animals comprising a human or humanized IL-4 gene under control of non-human IL-4 regulatory elements is also provided, including non-human animals that have a replacement of non-human IL-4-encoding sequence with human IL-4-encoding sequence at an endogenous non-human IL-4 locus. Non-human animals comprising a human or humanized IL-4R? gene under control of non-human IL-4R? regulatory elements is also provided, including non-human animals that have a replacement of non-human IL-4R?-encoding sequence with human or humanized IL-4R?-encoding sequence at an endogenous non-human C IL-4R? locus.

Abstract: Some embodiments of the present disclosure relate to one or more compositions that upregulate the production of one or more sequences of mRNA. The sequences of mRNA may encode for translation of a target biomolecule, thereby causing an increase in bioavailability of the target biomolecule within a subject that is administered the one or more compositions. In some embodiments of the present disclosure, the target biomolecule is a protein, such as a toll-like receptor 9.

Abstract: Compositions, methods and uses of genetically modified NK cells to treat a patient with a tumor are presented. The genetically modified NK cells express a protein complex having an ? chain and a ? chain T cell receptor, at least a portion of which is specific to a patient- or tumor-specific neoepitope, or a tumor associated antigen, and at least a portion of CD3?, and at least a portion of CD3?. The genetically modified NK cells can be administered to a cancer patient to induce, maintain or augment a T cell immune response against the cancer or the tumor.

Abstract: Gene-modified, lymphoid-tissue-homing dendritic cells that comprise a 1-alpha-hydroxylase gene and a retinaldehyde dehydrogenase 2 gene, where the 1-alpha-hydroxylase gene is expressed to produce functional 1-alpha-hydroxylase enzyme and the retinaldehyde dehydrogenase 2 gene is expressed to produce functional retinaldehyde dehydrogenase 2 gene enzyme. A method for treating one or more than one inflammation-related condition or disease, the method comprising administering gene-modified, lymphoid-tissue-homing dendritic cells that comprise a 1-alpha-hydroxylase gene and a retinaldehyde dehydrogenase 2 gene, where the 1-alpha-hydroxylase gene is expressed to produce functional 1-alpha-hydroxylase enzyme and the retinaldehyde dehydrogenase 2 gene is expressed to produce functional retinaldehyde dehydrogenase 2 gene enzyme.

Abstract: Described herein are diagnostic and control fusion protein reagents and methods for use thereof in simple rapid and inexpensive hemagglutinin assays for the detection of subject antibodies directed to the SARS-CoV-2 virus.

Abstract: Provided for herein are viral particles comprising a heterologous viral glycoprotein and a targeting moiety, wherein the targeting moiety comprises a polypeptide comprising a formula of T-S1, wherein T is a target binding domain and S1 is a stalk portion. The stalk portion may comprise a variant Fe domain. The stalk portion may comprise a flexible polypeptide domain. The targeting moiety comprising the formula T-S1 may be incorporated into a viral particle to assist with targeting such particles to a specific cell type. Also provided for herein are compositions comprising the same, and methods of using the same.