Institut National De La Sante Et De La Recherche Medicale

Centre National De La Recherche Scientifique

Assistance Publique - Hopitaux De Paris (Aphp)

Cornell University

Universitéde Strasbourg

Commissariat a L'energie Atomique Et Aux Energies Alternatives

Universitè De Montpellier

Université Paris Descartes

Université Paris—sud

Université Paris Sud Xi

Universite Evry Val D'essone

Patrick Aubourg

Jean-Louis Mandel

Jean Mosser

Claude Sarde

Ronald G Crystal

Helene Monique Puccio

Pierre Bougneres

Jocelyne Caboche

Nathalie Cartier-Lacave

Nicolas Tricaud

Nathalie Cartier

Benoît Gautier

Sandrine Betuing

Eloise Hudry

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.

Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject

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).

Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy due to Friedreich ataxia

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.

Expression vector for cholesterol 24-hydrolase in therapy of Huntington's disease

Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia

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.

CYP46A1 gene for the treatment of alzheimer's disease

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.

X-linked adrenoleukodystrophy gene and corresponding protein

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.

The cDNA and genomic DNA sequences encoding the human adrenoleukodystrophy protein are provided. Mutations of the adrenoleukodystrophy protein cause adrenoleukodystrophy or adrenomyelopathy.

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Le Kremlin Bicetre, France

Patrick Aubourg