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: 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 present invention relates to methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject. In particular, the present invention relates to a method for selectively expressing a polynucleotide of interest in the peripheral nervous system of a subject in need thereof comprising the step of transducing a peripheral nerve of the subject with an amount of an AVV9 vector containing the polynucleotide of interest.

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: The present invention relates to methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject. In particular, the present invention relates to a method for selectively expressing a polynucleotide of interest in the peripheral nervous system of a subject in need thereof comprising the step of transducing a peripheral nerve of the subject with an amount of an AVV9 vector containing the polynucleotide of interest.

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: The present invention relates to a vector for use in the treatment of Huntington's disease, which vector comprises a cholesterol 24-hydroxylase encoding nucleic acid.

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 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: The present invention relates to a vector for use in the treatment of Huntington's disease, which vector comprises a cholesterol 24-hydroxylase encoding nucleic acid.

Abstract: The invention relates to a viral vector for treating Alzheimers disease, which vector comprises a cholesterol 24-hydroxylase (CYP46A1) encoding nucleic acid. In a preferred embodiment, the viral vector may be an Adeno-Associated-Virus (AAV) vector, preferably an AVV5 vector. The vector may be useful for the manufacture of a pharmaceutical composition for the treatment of Alzheimers disease in a subject, wherein the vector is to be administered directly into the brain of the subject or by intravenous or intrathecal injection.

Abstract: The invention relates to a viral vector for treating Alzheimers disease, which vector comprises a cholesterol 24-hydroxylase (CYP46A1) encoding nucleic acid. In a preferred embodiment, the viral vector may be an Adeno-Associated-Virus (AAV) vector, preferably an AVV5 vector. The vector may be useful for the manufacture of a pharmaceutical composition for the treatment of Alzheimers disease in a subject, wherein the vector is to be administered directly into the brain of the subject or by intravenous or intrathecal injection.

Abstract: Provided is an assembly for insulating substantially vertical runs of cooled surfaces that are continuously or periodically exposed to air or another gas that has a dewpoint temperature above that of the cooled surface, thereby resulting in condensation water vapor on or adjacent the cooled surface. One embodiment of the insulating assembly provides a wicking material layer adjacent the cooled surface that, in combination with transporting and evaporating structures, removes the condensate from the vicinity of the cooled surface and allows for its evaporation into the ambient gas surrounding the exterior surface of the insulating assembly. Another embodiment provides for exposing the condensate to an internal conditioned gas stream to provide for evaporation that does not necessarily utilize an exterior surface. Both embodiments remove condensate from the insulating structure and thereby tend to avoid any substantial condensate accumulation and maintain the insulating performance.

Abstract: Provided is an assembly for insulating substantially vertical runs of cooled surfaces that are continuously or periodically exposed to air or another gas that has a dewpoint temperature above that of the cooled surface, thereby resulting in condensation water vapor on or adjacent the cooled surface. One embodiment of the insulating assembly provides a wicking material layer adjacent the cooled surface that, in combination with transporting and evaporating structures, removes the condensate from the vicinity of the cooled surface and allows for its evaporation into the ambient gas surrounding the exterior surface of the insulating assembly. Another embodiment provides for exposing the condensate to an internal conditioned gas stream to provide for evaporation that does not necessarily utilize an exterior surface. Both embodiments remove condensate from the insulating structure and thereby tend to avoid any substantial condensate accumulation and maintain the insulating performance.

Abstract: The present invention provides novel methods for delivering cells, particularly modified cells to the central nervous system (CNS). The purpose of this invention is to present a method that provides sustained delivery of a molecule to the central nervous system, thereby increasing the bioavailability of the molecule and lengthening the possible duration of treatment.

Abstract: The present invention relates generally to efficient delivery of viral vectors to cells of the CNS, particularly useful in the treatment of neurodegenerative disorders and motor neuron diseases. The invention involves selecting a first population and a second population of synaptically connected neurons, wherein a therapeutic polypeptide is to be expressed in said second population of neurons; and administering rAAV virions comprising a therapeutic gene to said first subpopulation of neurons of said subject such that the rAAV virions are transported across a synapse between synaptically connected neurons.

Abstract: The present invention relates to the use of riluzole or one of its pharmaceutically acceptable salts for the prevention and treatment of adrenoleukodystrophy.

Abstract: The present invention relates to the use of riluzole or one of its pharmaceutically acceptable salts for the prevention and treatment of adrenoleukodystrophy.

Abstract: The present invention relates to adrenoleukodystrophy proteins and uses thereof. More particularly, the invention relates to human adrenoleukodystrophy proteins. Mutations of the adrenoleukodystrophy protein cause adrenoleukodystrophy or adrenomyelopathy.

Abstract: A method for the treatment of adrenoleukodystrophy or adrenomyeloneuropathy, in which there is administered to a human patient in need of the same an effective amount of hematopoietic cells modified ex vivo by infection with a retroviral vector containing a nucleic acid fragment comprising a sequence as represented on FIG. 2 (SEQ. ID No: 1) or FIG. 6 (SEQ. ID. No: 4-17) or FIG. 7 (SEQ. ID No: 18-23). Also, human hematopoietic cells can be treated ex vivo to correct the content of very long chain fatty acids in them, by providing human hematopoietic cells of a patient affected by adrenoleukodystrophy or adrenomyeloneuropathy, and infecting them ex vivo with a retroviral vector containing, operably linked to a promoter, a nucleic acid fragment as above. The expression of the nucleic acid in the hematopoietic cells corrects the content of very long chain fatty acids in the hematopoietic cells.