Genethon

Évry, France
Évry, France
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Patent
Genethon and Wake forest University | Date: 2017-02-22

A composition comprising a molecule for use in the delivery of the molecule to the peripheral nervous system (PNS) and/or to the central nervous system (CNS), wherein the composition is administered by regional infusion.


The present invention relates to methods for the production of biopharmaceuticals implementing a baculovirus-based system. These methods advantageously allow the production of biopharmaceuticals with a reduced number of or without contaminating baculoviral virions.


Patent
Genethon and Wake forest University | Date: 2015-04-20

A composition comprising a molecule for use in the delivery of the molecule to the peripheral nervous system (PNS) and/or to the central nervous system (CNS), wherein the composition is administered by regional infusion.


Patent
Genethon and International Center For Genetic Engineering And Biotechnology | Date: 2015-04-27

The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.


Patent
French Institute of Health, Medical Research, French National Center for Scientific Research, Genethon, University of Paris Descartes, École Nationale Supérieure de Chimie de Paris, University of Évry Val d'Essonne and Assistance Publique Hopitaux De Paris | Date: 2016-09-16

The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA


Patent
Genethon and Royal Holloway And Bedford New College | Date: 2015-04-24

A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein:the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin;the composition is systemically administered.


Patent
Wake forest University, Genethon and Childrens Medical Center Corporation | Date: 2016-07-26

The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.


Patent
Genethon and Icgeb International Center For Genetic Engineering And Biotechnology | Date: 2017-03-01

The invention relates to a nucleic acid sequence useful in the treatment of hyperbilirubinemia, in particular in the treatment of Crigler-Najjar syndrome. More particularly, the nucleic acid sequence of the present invention is a codon-optimized UGT1A1 coding sequence.


Patent
Wake forest University, Genethon and Childrens Medical Center Corporation | Date: 2016-12-20

The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.


A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein:- the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin;- the composition is systemically administered.

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