Abstract: The present disclosure provides a method of generating a stable producer cell line. The generation of stable producer cell lines, such as those provided in accordance with the present invention, increases the reproducibility and ease of creating high titer lentiviral stocks while easing biosafety concerns and the variation in expressed envelope proteins defines the tropism of the generated virus. The present disclosure also provides for a novel lentiviral transfer vector plasmid.

Abstract: Methods and compositions for treating symptoms of conditions such as but not limited to osteoarthritis and rheumatoid arthritis. The methods may feature direct intraarticular injection of a recombinant self-complementary adeno-associated virus (sc-rAAV) with a vector adapted to express a modified IL-1Ra peptide. The methods of the present invention may express a therapeutically effective amount of the modified IL-1Ra peptide so as to ameliorating symptoms associated with the condition being treated.

Abstract: The present disclosure provides a method of generating a stable producer cell line. The generation of stable producer cell lines, such as those provided in accordance with the present invention, increases the reproducibility and ease of creating high titer lentiviral stocks while easing biosafety concerns and the variation in expressed envelope proteins defines the tropism of the generated virus. The present disclosure also provides for a novel lentiviral transfer vector plasmid.

Abstract: The disclosed methods are generally directed to preventing, treating, suppressing, controlling or otherwise mitigating side effects of T-cell therapy, the T-cell therapy designed to accelerate immune reconstitution, induce a graft-versus-malignancy effect, and/or target tumor cells. In some embodiments, the present disclosure provides expression vectors including a first expression control sequence operably linked to a first nucleic acid sequence, the first nucleic acid sequence encoding a shRNA to knockdown hypoxanthine-guanine phosphoribosyl transferase.

Abstract: The present disclosure is directed to polymer nanocapsules including a ribonucleoprotein complex, and methods of their delivery. In some embodiments, the polymer nanocapsule comprises a polymer shell and a ribonucleoprotein complex, wherein the polymer shell comprises at least one positively charged monomer, at least one neutral monomer, and a crosslinker. Also disclosed are conjugates of polymer nanocapsules coupled to one or more targeting moieties and/or one or more stabilizing moieties.

Abstract: Methods and compositions for treating symptoms of conditions such as but not limited to osteoarthritis and rheumatoid arthritis in canines. The methods may feature direct intraarticular injection of a recombinant self-complementary adeno-associated virus (sc-rAAV) with a vector adapted to express a modified IL-1Ra peptide. The methods of the present invention may express a therapeutically effective amount of the modified IL-1Ra peptide so as to ameliorating symptoms associated with the condition being treated.

Abstract: In one aspect of the present disclosure is a method of harvesting viral titer about every 40 hours to about every 56 hours following induction of stable producer cell line cells, wherein the viral titer is at least partially harvested in a serum-free medium. In another aspect of the present disclosure is a method of harvesting vector supernatant comprising: generating stable producer cell line cells; inducing viral vector production from the generated stable producer cell line cells; and repeatedly harvesting the viral vectors from the induced generated stable producer cell line cells in serum-free media every about 40 to about 56 hours following an initial harvesting of the viral vectors.

Abstract: Methods and compositions for treating symptoms of conditions such as but not limited to osteoarthritis in horses. The methods may feature direct intraarticular injection of a recombinant self-complementary adeno-associated virus (sc-rAAV) with a vector adapted to express a modified IL-1 Ra peptide. The methods of the present invention may express a therapeutically effective amount of the modified IL-1 Ra peptide so as to ameliorating symptoms associated with the condition being treated.

Abstract: Methods and compositions for treating symptoms of conditions such as but not limited to osteoarthritis and rheumatoid arthritis. The methods may feature direct intraarticular injection of a recombinant self-complementary adeno-associated virus (sc-rAAV) with a vector adapted to express a modified IL-1Ra peptide. The methods of the present invention may express a therapeutically effective amount of the modified IL-1Ra peptide so as to ameliorating symptoms associated with the condition being treated.

Abstract: The present disclosure is directed to methods of selecting for modified stem cells in vivo. The present disclosure is also directed to a 6TG dosing schedule. The present disclosure is also directed to an oral formulation comprising 6TG.

Abstract: The present disclosure provides a method of generating a stable producer cell line. The generation of stable producer cell lines, such as those provided in accordance with the present invention, increases the reproducibility and ease of creating high titer lentiviral stocks while easing biosafety concerns and the variation in expressed envelope proteins defines the tropism of the generated virus. The present disclosure also provides for a novel lentiviral transfer vector plasmid.

Abstract: The present disclosure provides a method of generating a stable producer cell line. The generation of stable producer cell lines, such as those provided in accordance with the present invention, increases the reproducibility and ease of creating high titer lentiviral stocks while easing biosafety concerns and the variation in expressed envelope proteins defines the tropism of the generated virus. The present disclosure also provides for a novel lentiviral transfer vector plasmid.

Abstract: The present disclosure is directed to methods of selecting for modified stem cells in vivo. The present disclosure is also directed to a 6TG dosing schedule. The present disclosure is also directed to an oral formulation comprising 6TG.

Abstract: The present disclosure provides a method of generating a stable producer cell line. The generation of stable producer cell lines, such as those provided in accordance with the present invention, increases the reproducibility and ease of creating high titer lentiviral stocks while easing biosafety concerns and the variation in expressed envelope proteins defines the tropism of the generated virus. The present disclosure also provides for a novel lentiviral transfer vector plasmid.

Abstract: The invention relates to Adeno-associated virus vectors. In particular, it relates to Adeno-associated virus vectors with modified capsid proteins and materials and methods for their preparation and use.

Abstract: The invention relates to Adeno-associated virus vectors. In particular, it relates to Adeno-associated virus vectors with modified capsid proteins and materials and methods for their preparation and use.

Abstract: The invention relates to Adeno-associated virus vectors. In particular, it relates to Adeno-associated virus vectors with modified capsid proteins and materials and methods for their preparation and use.

Abstract: The invention relates to Adeno-associated virus vectors. In particular, it relates to Adeno-associated virus vectors with modified capsid proteins and materials and methods for their preparation and use.

Abstract: A computer system having a CPU, a non-volatile storage device, a power management processor having a volatile power management configuration, and a power supply in circuit communication. Responsive to the AC power to the power supply being interrupted, the CPU restores the volatile power management configuration to said power management processor. The power management configuration comprises a value corresponding to a wake alarm and whether the power management processor responds to external events.