Abstract: Compositions and methods to prevent, inhibit or treat a disease or disorder associated with expression of APOE4 in a mammal are provided.

Abstract: Modified AAV vectors and uses thereof are provided.

Abstract: Compositions having nucleic acid sequences for regulating expression from gene therapy vectors, and methods of using the compositions, are provided.

Abstract: A gene therapy vector comprising an expression cassette coding for a mammalian apolipoprotein E that has a residue other than arginine at at least one of positions 112, 136, or 158, but is not a mammalian apolipoprotein E that has R112, R136 and R158 or a mammalian apolipoprotein E that has C112, R136 and C158, or coding for an antibody that binds to APOE4 or disrupts the binding of APOE to heparan sulfate proteoglycans, and methods of using the vector, are provided.

Abstract: A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).

Abstract: Provided is a method of treating coronary artery disease in a mammal, comprising administering to a region of the heart of the mammal (a) a first vector encoding one or more angiogenic proteins which induce vascularization in the heart of the mammal, and (b) a second vector encoding one or more cardio-differentiating transcription factors which induce the production of induced cardiomyocytes (iCM) in the heart of the mammal, whereby the coronary artery disease in the mammal is treated. In a preferred embodiment, the first vector is an adenoviral vector encoding VEGF and the second vector is a lentiviral vector encoding Gata4, Mef2c, and Tbx5 (GMT).

Abstract: Compositions and methods for anti-oxidant therapy are provided.

Abstract: The present invention relates to a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure. More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.

Abstract: The invention provides methods to inhibit or treat brain cancers by locally inhibiting expression or activity of growth factors or growth factor receptors.

Abstract: The invention provides a method of treating ovarian cancer in a patient by administering to the patient an adeno-associated virus (AAV) vector encoding an anti-VEGF antibody or antigen binding fragment thereof.

Abstract: Compositions and methods for eosinophilia in a mammal are provided. In one embodiment, the composition is a viral gene therapy vector, and a single dose of the vector reduces increased lumbers of eosinophils in a mammal.

Abstract: Gene therapy compositions and methods to inhibit or treat neurodegenerative diseases, e.g., Alzheimer's disease, are provided.

Abstract: A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).

Abstract: A gene therapy treatment for alpha 1-antitrypsin (AAT) deficiency is provided comprising a plasmid or viral, e.g., an AAV, vector coding for an elastase- or cathepsin G-inhibiting, oxidation-resistant human AAT with a substitution at, for example, Met358 and/or Met351.

Abstract: Modified AAV vectors and uses thereof are provided.

Abstract: Provided is a method of treating coronary artery disease in a mammal, comprising administering to a region of the heart of the mammal (a) a first vector encoding one or more angiogenic proteins which induce vascularization in the heart of the mammal, and (b) a second vector encoding one or more cardio-differentiating transcription factors which induce the production of induced cardiomyocytes (iCM) in the heart of the mammal, whereby the coronary artery disease in the mammal is treated. In a preferred embodiment, the first vector is an adenoviral vector encoding VEGF and the second vector is a lentiviral vector encoding Gata4, Mef2c, and Tbx5 (GMT).

Abstract: Provided is a method of treating coronary artery disease in a mammal, comprising administering to a region of the heart of the mammal (a) a first vector encoding one or more angiogenic proteins which induce vascularization in the heart of the mammal, and (b) a second vector encoding one or more cardio-differentiating transcription factors which induce the production of induced cardiomyocytes (iCM) in the heart of the mammal, whereby the coronary artery disease in the mammal is treated. In a preferred embodiment, the first vector is an adenoviral vector encoding VEGF and the second vector is a lentiviral vector encoding Gata4, Mef2c, and Tbx5 (GMT).

Abstract: A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).

Abstract: A vector comprising a promoter operably linked to a nucleic acid sequence encoding human aldehyde dehydrogenase, as well as a composition comprising the vector and method of using the vector to treat aldehyde dehydrogenase deficiency, or to prevent or treat a disease characterized by aldehyde dehydrogenase deficiency.

Abstract: This invention is directed to a vector which comprises a promoter operably linked to a nucleic acid sequence encoding a therapeutic gene that blocks allergic reactions. The invention is also directed to a composition comprising the vector and method of using the vector to reduce or inhibit an immune response or allergic reaction to an allergen in a mammal.