Abstract: The present invention relates to a method for purifying a Factor VIII (FVIII) subspecies from a composition comprising FVIII, said method comprising an anion exchange chromatography step, a size exclusion chromatography step, and a concentration step. The invention also relates to a composition comprising a purified FVIII subspecies.

Abstract: The present specification discloses methods of characterizing a population of particles using a particle analyzer. Particles may be characterized by size, absolute number, whether the particle is non-proteinaceous or proteinaceous, whether a proteinaceous particle has or lacks a certain physical property, or any combination thereof.

Abstract: The present invention relates to a method for purifying a Factor VIII (FVIII) subspecies from a composition comprising FVIII, said method comprising an anion exchange chromatography step, a size exclusion chromatography step, and a concentration step. The invention also relates to a composition comprising a purified FVIII subspecies.

Abstract: The present invention relates to a method for purifying a Factor VIII (FVIII) subspecies from a composition comprising FVIII, said method comprising an anion exchange chromatography step, a size exclusion chromatography step, and a concentration step. The invention also relates to a composition comprising a purified FVIII subspecies.

Abstract: Methods are provided for treating a patient with viral-based gene therapy that promote persistent transgene expression. The methods include administering to the patient an inhibitor of the interleukin-6 (IL6) signaling pathway or the NCOR2/SMRT histone deacetylation pathway, and a viral-based gene therapy vector. Methods are also provided for assigning viral-based gene therapy to a patient that include determining whether the patient has a genotype sensitizing the patient to persistent infection by a viral-based gene therapy vector by evaluating whether the patient has a mutation in the SMRT/NCOR2 gene associated with reduced SMRT/NCOR2 protein function or in the interleukin-6 receptor (IL-6R) gene associated with reduced IL-6R function. If the patient has either a mutation in the SMRT/NCOR2 gene associated with reduced SMRT/NCOR2 protein function or a mutation in the IL-6R gene associated with reduced IL-6R function, a viral-based gene therapy vector is assigned to the patient.

Abstract: The present specification discloses methods of characterizing a population of particles using a particle analyzer. Particles may be characterized by size, absolute number, whether the particle is non-proteinaceous or proteinaceous, whether a proteinaceous particle has or lacks a certain physical property, or any combination thereof.

Abstract: The present specification discloses methods of characterizing a population of particles using a particle analyzer. Particles may be characterized by size, absolute number, whether the particle is non-proteinaceous or proteinaceous, whether a proteinaceous particle has or lacks a certain physical property, or any combination thereof.

Abstract: The present invention relates to a method for purifying a Factor VIII (FVIII) subspecies from a composition comprising FVIII, said method comprising an anion exchange chromatography step, a size exclusion chromatography step, and a concentration step. The invention also relates to a composition comprising a purified FVIII subspecies.

Abstract: The present invention relates to a cell-based in vitro screening assay for identifying and selecting therapeutic agents that inhibit amyloid ?-induced cytotoxicity.

Abstract: The present invention relates, in general, to development of non-human transgenic animals expressing a human blood clotting factor, such as Factor VIII, Factor VII, Factor IX and von Willebrand factor. The invention further provides methods of detecting immunogenic events against human blood clotting factor using the transgenic animals described.

Abstract: The present invention provides methods for providing a prognosis of treatment of diseases associated with inflammatory disease of the brain, including MS, e.g., relapsing-remitting multiple sclerosis (RRMS), Alzheimer's disease, and Parkinson's disease using molecular markers that are shown to be overexpressed or underexpressed in patients treated with intravenous immunoglobulins (IVIG). Also provided are methods to identify compounds that are useful for the treatment or prevention of MS, e.g., relapsing-remitting multiple sclerosis (RRMS), Alzheimer's disease, and Parkinson's disease.

Abstract: The present invention provides methods for providing a prognosis of treatment of diseases associated with inflammatory disease of the brain, including MS, e.g., relapsing-remitting multiple sclerosis (RRMS), Alzheimer's disease, and Parkinson's disease using molecular markers that are shown to be overexpressed or underexpressed in patients treated with intravenous immunoglobulins (IVIG). Also provided are methods to identify compounds that are useful for the treatment or prevention of MS, e.g., relapsing-remitting multiple sclerosis (RRMS), Alzheimer's disease, and Parkinson's disease.

Abstract: The present invention relates, in general, to development of non-human transgenic animals expressing a human blood clotting factor, such as Factor VIII, Factor VII, Factor IX and von Willebrand factor. The invention further provides methods of detecting immunogenic events against human blood clotting factor using the transgenic animals described.

Abstract: The present invention relates to a cell-based in vitro screening assay for identifying and selecting therapeutic agents that inhibit amyloid r-induced cytotoxicity.

Abstract: The present invention provides methods for providing a prognosis of treatment of diseases associated with inflammatory disease of the brain, including MS, e.g., relapsing-remitting multiple sclerosis (RRMS), Alzheimer's disease, and Parkinson's disease using molecular markers that are shown to be overexpressed or underexpressed in patients treated with intravenous immunoglobulins (IVIG). Also provided are methods to identify compounds that are useful for the treatment or prevention of MS, e.g., relapsing-remitting multiple sclerosis (RRMS), Alzheimer's disease, and Parkinson's disease.