Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.

Abstract: The present invention relates to nucleic acid expression cassettes that are engineered to enhance gene expression. Vectors and methods employing the expression cassettes containing novel chimeric regulatory elements are provided. The invention is particularly useful for delivery of transgenes to target cells and confers desirable properties for liver-directed and muscle-gene therapy. Moreover, the invention relates to gene therapy methods of delivery of therapeutics for treating various disorders.

Abstract: Provided are methods of treating or ameliorating the symptoms of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy by administration of therapeutically effective doses of recombinant adeno-associated viruses (rAAV) containing a transgene encoding AUF1 and a second rAAV encoding a microdystrophin or other therapeutic effective to treat the dystrophinopathy. Also provided are rAAV vectors encoding AUF1 proteins.

Abstract: Provided are methods and compositions for the delivery of fully human post-translationally modified therapeutic TNF receptor Fc fusions. The fully human post-translationally modified therapeutic TNF receptor Fc fusions may be delivered by gene therapy methods, e.g., as a recombinant adeno-associated virus (rAAV) vector to the appropriate tissue. Also provided are methods of treating ocular indications such as non-infectious uveitis with the fully human post-translationally modified therapeutic TNF receptor Fc fusions.

Abstract: Provided are methods of treating or ameliorating the symptoms of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy by administration of therapeutically effective doses of recombinant adeno-associated viruses (rAAV) containing a transgene encoding a microdystrophin in combination with a second therapeutic which is effective to treat or ameliorate the symptoms of the dystrophinopathy.

Abstract: The present invention relates to nucleic acid expression cassettes that are engineered to express Tau-specific antibodies. Vectors and methods of employing the expression cassettes containing novel Tau-specific antibodies are provided. The invention is particularly useful for delivery of transgenes to target CNS cells and confers desirable properties for CNS-directed gene therapy. Moreover, the invention relates to a novel method of engineering, and expressing Tau-specific antibody transgenes for example within CNS cells, and delivery of therapeutics for treating various tauopathic diseases and disorders.

Abstract: The present invention relates to nucleic acid expression cassettes that are engineered to include polynucleotide sequences useful as inert stuffer (or filler) sequences in expression cassettes, particularly useful for transgene expression delivered as viral vectors, incorporating the engineered expression cassettes described herein, including rAA Vs, for use in therapy.

Abstract: Provided are methods of treating or ameliorating the symptoms of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy by administration of therapeutically effective doses of recombinant adeno-associated viruses (rAAV) containing a transgene encoding a microdystrophin.

Abstract: Compositions and methods are described for the delivery of a fully human post-translationally modified therapeutic monoclonal antibody that binds to CGRP or CGRP receptor to a human subject for the treatment or prevention of migraines and cluster headaches. The antibodies may be delivered by gene therapy vectors, particularly rAAV vectors. Also provided are dual transgene constructs for the delivery of anti-CGRP and anti-CGRP receptor antibodies or antigen binding fragments thereof.

Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.

Abstract: Compositions and methods are described for the delivery of a fully human post-translationally modified therapeutic monoclonal antibody, or an antigen binding fragment thereof, that binds to TNF-?, IL6 or IL6-R to a human subject for treatment of an ocular indication, particularly non-infectious uveitis. The nucleotide sequence encoding the antibody is delivered in a rAAV vector that targets ocular tissue cells for expression of the transgene.

Abstract: Provided is an invention based, in part, on novel gene constructs that encode a microdystrophin protein for use in gene therapy. The microdystrophin gene constructs and expression cassettes were engineered for improved therapy with respect to efficacy, potency and safety to the subject when expressed by a viral vector in muscle cells and/or CNS cells.

Abstract: The present invention relates to nucleic acid expression cassettes that are engineered to enhance gene expression. Vectors and methods employing the expression cassettes containing novel chimeric regulatory elements are provided. The invention is particularly useful for delivery of transgenes to target cells and confers desirable properties for liver-directed and muscle-directed or liver-directed and bone-directed gene therapy. Moreover, the invention relates to a novel method of engineering tandem enhancer/promoter elements and expressing transgenes for example within liver and/or muscle cells, and delivery of therapeutics for treating various disorders.

Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.

Abstract: Provided are methods and compositions for the delivery of fully human post-translationally modified therapeutic monoclonal antibodies and antigen-binding fragments thereof. The fully human post-translationally modified therapeutic monoclonal antibodies may be delivered by gene therapy methods, e.g., as a recombinant adeno-associated vims (rAAV) vector to the appropriate tissue. Methods of manufacture of the AAV vectors, pharmaceutical compositions and methods of treatment are also provided. In addition, provided are methods of producing therapeutic antibodies that are “biobetters” as fully human post-translationally modified. These fully human post-translationally modified therapeutic antibodies may be administered to a subject in need of treatment with the therapeutic antibody.

Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.

Abstract: This invention relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same, and methods of using the same for delivery of mini-dystrophin to a cell or a subject.