Abstract: The present invention is directed to substituted pyrazolo[1,5-a]pyridines and related methods for their synthesis and use.

Abstract: Methods of antagonizing MIF activity using ibudilast are described. Also described are methods of screening for MIF antagonists. These agents can be used for treating addictions, including drug and behavioral addictions, as well as for treating neuropathic pain.

Abstract: The present disclosure is directed to methods for providing tolperisone having extremely low levels of 4-MMPPO (2-methyl-1-(4-methylphenyl)-propenone), as well as related compositions. The invention further relates to methods of treating a subject with tolperisone under conditions that limit exposure of the subject to tolerable levels of 4-MMPPO, among other features.

Abstract: The present invention is directed to substituted pyrazolo[1,5-a]pyridines and related methods for their synthesis and use.

Abstract: The present invention is directed to the use of ibudilast for treating neuropathic pain.

Abstract: Methods for treating thrombotic disorders using sulfated polysaccharides such as fucoidans are disclosed.

Abstract: The present invention is directed to substituted pyrazolo[1,5-?]pyridines and related methods for their synthesis and use.

Abstract: The present invention is directed to the use of ibudilast for treating neuropathic pain.

Abstract: AAV expression vectors and recombinant virions produced using these vectors, which include genes coding for enzymes defective or missing in lysosomal storage disorders, are described. These recombinant AAV virions are useful in the treatment of a variety of lysosomal storage disorders and the methods described herein provide for long-term, sustained expression of the defective or missing enzyme.

Abstract: The present invention provides methods and compositions for producing high titer, wild-type-free preparations of recombinant AAV (“rAAV”) virions. The compositions of the present invention include novel nucleic acids encoding AAV helper functions and AAV helper function vectors. The present invention also includes host cells transfected by the claimed nucleic acids, methods of using the claimed vectors, and rAAV virions produced by such methods.

Abstract: The present invention provides methods and compositions for producing high titer, wild-type-free preparations of recombinant AAV (“rAAV”) virions. The compositions of the present invention include novel nucleic acids encoding AAV helper functions and AAV helper function vectors. The present invention also includes host cells transfected by the claimed nucleic acids, methods of using the claimed vectors, and rAAV virions produced by such methods.

Abstract: Vectors and methods are provided for treatment of neurodegenerative disorders by administration of anti-inflammatory cytokines, such as IL-10. Anti-inflammatory cytokines can be administered as a protein or by gene therapy, using plasmid delivery or a viral vector such as adeno-associated virus (AAV). Diseases including Parkinson's disease, Amyotrophic Lateral Sclerosis, Alzheimer's disease and Multiple Sclerosis may be treated using the vectors and methods of the invention.

Abstract: Compositions and methods are provided for preparation of concentrated stock solutions of AAV virions without aggregation. Formulations for AAV preparation and storage are high ionic strength solutions (e.g. ?˜500 mM) that are nonetheless isotonic with the intended target tissue. This combination of high ionic strength and modest osmolarity is achieved using salts of high valency, such as sodium citrate. AAV stock solutions up to 6.4×1013 vg/mL are possible using the formulations of the invention, with no aggregation being observed even after ten freeze-thaw cycles. The surfactant Pluronic® F68 may be added at 0.001% to prevent losses of virions to surfaces during handling. Virion preparations can also be treated with nucleases to eliminate small nucleic acid strands on virions surfaces that exacerbate aggregation.

Abstract: Methods of making and using recombinant AAV vectors and virions for gene delivery to the lung are described. The recombinant AAV virions are derived from caprine AAV and bovine AAV, both of which display tropism for lung tissue.

Abstract: The present invention provides methods and compositions for producing high titer preparations of recombinant AAV (“rAAV”) virions. The compositions of the present invention include AAV helper function systems and host cells. The present invention also includes methods of using AAV helper function vectors that effect the production of only small amounts of the long forms of Rep protein, and rAAV virions produced by such methods.

Abstract: Methods for treating bleeding disorders using non-anticoagulant sulfated polysaccharides (NASPs) as procoagulants are disclosed. NASPs can be administered as single agents, or in combination with one another, or with other medications (such as factors VII, VIII and IX) to promote hemostasis. In particular, the use of NASPs in treatment of bleeding disorders, including congenital coagulation disorders, acquired coagulation disorders, and trauma induced hemorrhagic conditions is described.

Abstract: Methods of delivering viral vectors, particularly recombinant AAV virions, to the CNS are provided. Also provided are methods of treating Parkinson's Disease.

Abstract: Methods of delivering viral vectors, particularly recombinant AAV virions, to the CNS are provided. Also provided are methods of treating Parkinson's Disease.

Abstract: The present invention provides compositions and methods of producing recombinant AAV (rAAV) virions in large amounts or high titers. Also provided are methods for producing stably transformed host cells capable of producing rAAV virions.

Abstract: Accessory functions capable of supporting efficient recombinant AAV (rAAV) virion production in a suitable host cell are provided. The accessory functions are in the form of one or more vectors that are capable of being transferred between cells. Methods of producing rAAV virions are also provided. The methods can be practiced to produce commercially significant levels of rAAV particles without also generating significant levels of infectious helper virus or other contaminating by-products.