GENETHON Patent applications |
Patent application number | Title | Published |
20150241452 | METHOD FOR DIAGNOSING MUSCULAR DYSTROPHY - The invention relates to a method for the diagnosis, prognosis and therapeutic monitoring of muscular dystrophy, by detecting titin or one or more fragments of titin in a bodily fluid of a patient. | 08-27-2015 |
20150141361 | USE OF EPIGENOME-MODIFYING COMPOUNDS FOR THE TREATMENT OF GENETIC MUSCULAR DISEASES LINKED TO A PROTEIN-CONFORMATIONAL DISORDER - A pharmaceutical composition including at least one epigenome-modifying compound, for a use thereof in the treatment of genetic muscular diseases linked to a conformational disorder of at least one protein, said disorder causing the cellular degradation of the protein. | 05-21-2015 |
20150111951 | METHODS FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS - 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 | 04-23-2015 |
20140356904 | BACULOVIRUS EXPRESSION SYSTEMS - The present invention relates to an optimized baculovirus construct useful for the production of virus (-like) particles or viral vectors, in particular viral vectors for gene therapy. | 12-04-2014 |
20140349374 | BACULOVIRUS SYSTEM FOR THE EXPRESSION OF A GENE THERAPY VECTOR - The invention concerns a recombinant baculovirus genome useful for the expression of gene therapy vectors by means of a single infection. | 11-27-2014 |
20140255349 | PEPTIDES WITH VIRAL INFECTION ENHANCING PROPERTIES AND THEIR USE - The invention relates to peptides and functional derivatives thereof and their use for improving transduction efficiency of viruses into target cells. | 09-11-2014 |
20140249211 | Systemic Gene Replacement Therapy for Treatment of X-Linked MyoTubular Myopathy (XLMTM) - 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. | 09-04-2014 |
20140179767 | METHODS FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS - 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 | 06-26-2014 |
20130189225 | METHODS OF INCREASING EFFICIENCY OF VECTOR PENETRATION OF TARGET TISSUE - Methods for increasing the efficiency of target tissue penetration of an adeno-associated virus (AAV) vector in a patient are provided. In some aspects, the methods involve inhibiting the interaction of the serum protein galectin 3 binding protein (G3BP) with AAV vector. Further provided are methods for reducing tissue distribution of a virus or for neutralizing a virus harbored by an organ destined for transplant, or newly transplanted, by administering a composition comprising G3BP. | 07-25-2013 |
20130171172 | CALPAIN-3 INHIBITORS FOR TREATING MUSCULAR DYSTROPHIES AND CARDIOMYOPATHIES - A composition comprising a calpain-3 inhibitor for treating muscular dystrophies and cardiomyopathies, in particular tibial muscular dystrophy (TMD). | 07-04-2013 |
20130072541 | ADENO-ASSOCIATED VIRAL VECTOR FOR EXON SKIPPING IN A GENE ENCODING A DISPENSIBLE-DOMAIN PROTEIN - The invention concerns an adeno-associated viral vector comprising:
| 03-21-2013 |
20130035353 | DRUGS FOR THE TREATMENT OF SARCOGLYCANOPATHIES - Inhibitors of the endoplasmic reticulum associated degradation (ERAD) pathway, particularly inhibitors of mannosidase I, are used for the preparation of a medicinal product intended to treat sarcoglycanopathies. | 02-07-2013 |
20120149010 | BACULOVIRUS-BASED PRODUCTION OF BIOPHARMACEUTICALS FREE OF CONTAMINATING BACULOVIRAL VIRIONS - 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 reduced or no contaminating baculoviral virions. | 06-14-2012 |
20120141422 | WIDESPREAD GENE DELIVERY TO THE RETINA USING SYSTEMIC ADMINISTRATION OF AAV VECTORS - The present invention relates to compositions and methods, in particular to methods based on systemic administration of scAAV, for delivering genes to cells of the retina of mammals, and in particular to photoreceptor cells, ganglion cells, glial cells, inner nuclear layer cells or cells of the retinal pigmented epithelium. | 06-07-2012 |
20120077860 | Adeno-Associated Viral Vector for Exon Skipping in a Gene Encoding a Dispensable Domain Protein - The invention concerns an adeno-associated viral vector comprising:
| 03-29-2012 |
20100266551 | ADENO-ASSOCIATED VIRAL VECTORS FOR THE EXPRESSION OF DYSFERLIN - The present invention relates to a composition comprising: a first adeno-associated viral (AAV) vector comprising: i) a 5′ITR (Inverted Terminal Repeat) sequence of AAV; ii) a portion of gene placed under the control of a promoter; iii) a sequence comprising a splice donor site; iv) a 3′ITR sequence of AAV; and/or a second adeno-associated viral (AAV) vector comprising; v) a 5′ITR (Inverted Terminal Repeat) sequence of AAV; vi) a sequence comprising a splice acceptor site; vii) a portion of gene; viii) a 3′ITR sequence of AAV. The combination of the portions of gene carried by the first and second AAV vectors comprises an open reading frame which encodes a functional dysferlin. In addition, the combination of the sequence comprising the splice donor site and the sequence comprising the splice acceptor site contains all the elements necessary for the splicing, advantageously derived from a natural intron of the dysferlin gene. | 10-21-2010 |
20100010036 | DRUGS FOR THE TREATMENT OF SARCOGLYCANOPATHIES - Inhibitors of the endoplasmic reticulum associated degradation (ERAD) pathway, particularly inhibitors of mannosidase I, are used for the preparation of a medicinal product intended to treat sarcoglycanopathies. | 01-14-2010 |
20090220468 | Specific CD4+CD25+ Regulatory T Cells for Haematopoietic Cell Transplantation and Immune Tolerance - A pharmaceutical composition comprising CD4+ CD25+ regulatory T cells specific for at least one minor histocompatibility antigen, and stem cells, advantageously haematopoietic, carrying at least the antigen can be used as a medicament for increasing the immune tolerance of a histocompatible host. | 09-03-2009 |