Abstract: 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. 2991+1655 A>G CEP290 mRNA.

Abstract: The present invention relates to gelling compositions, which changes from liquid state to gel state in function of temperature comprising: at least a poloxamer or mixture of poloxamers; at least a gelling agent; and at least an anticancer agent. Said compositions are advantageously used for local administration of an anticancer agent. Said compositions are useful for size-reduction of a tumour before surgical removal of said tumour, for preventing tumour recurrence after surgical removal of a tumour, and/or treating small tumours. They are therefore useful for the treatment of cancer, preferably a cancer of a wall of the digestive system or a gynaecologic cancer. The present invention also relates to a method for preparing said gelling compositions.

Abstract: The present invention relates to an optical imaging agent of formula (SIGNAL)n-SUPPORT-(L-BIOVECTOR)m, wherein: SUPPORT represents a physiologically acceptable chemical or biological substrate, with a particle size of between 1 and 100 nm, SIGNAL is a fluorophore, L is a linker of formula —C(X)—R1—Y—, with X being O, NH or S R1 being a (C1-C6)alkyl group, optionally R1 being a (C1-C6)alkyl group, optionally interrupted by 1 to 3 groups selected from —O—, —NH—, —C(O)—, —NHC(O)—, —(O)CNH—, —C(O)NH—N?C—, —N?C—, and (I), Y being NH or a —(O)CNH— group, and BIOVECTOR is a carbohydrate targeting markers of inflammation, advantageously selected from the group consisting of mannose, sialyl LewisX and derivatives thereof, n is greater than or equal to 0.5 and is less than 2, m is between 0 and 30, preferably between 1 and 30, more preferably between 5 and 20, diagnostic compositions comprising same and use thereof as a diagnostic agent (in vivo or ex vivo), or as contrast agent for image-guided surgery.

Abstract: 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. 2991+1655 A>G CEP290 mRNA.

Abstract: 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.2991+1655 A>G CEP290 mRNA.

Abstract: The present invention relates to gelling compositions, which changes from liquid state to gel state in function of temperature comprising: at least a poloxamer or mixture of poloxamers; at least a gelling agent; and at least an anticancer agent. Said compositions are advantageously used for local administration of an anticancer agent. Said compositions are useful for size-reduction of a tumour before surgical removal of said tumour, for preventing tumour recurrence after surgical removal of a tumour, and/or treating small tumours. They are therefore useful for the treatment of cancer, preferably a cancer of a wall of the digestive system or a gynaecologic cancer. The present invention also relates to a method for preparing said gelling compositions.

Abstract: Multimodal optical and magnetic resonance imaging methods based on the use of persistent luminescence nanoparticles. Use of mesoporous persistent luminescence <<core-shell>> complexes for theranostic applications.

Abstract: 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.

Abstract: The present invention relates to 4-azapodophyllotoxin analogs of formula (I) in which X, R1, R2, R3, R4 and Ar are as defined in claim 1, preferably a pharmaceutically acceptable salt thereof, optionally in the form of a solvate, a composition comprising said analogs, their use as medicament, in particular for the treatment of cancer, and a process for their preparation.

Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring 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.

Abstract: The invention relates to a composition including (1) an anionic macromolecule except nucleic acids, (2) a cationic lip, and (3) a nucleic acid having a size lower than or equal to 200 nucleotides, such as an interference RNA, and to the use thereof in gene therapy.

Abstract: 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.

Abstract: The present invention relates to specific regions of the N-terminal portion of the VAR2CSA protein and to the use of such specific regions in the prevention of pregnancy-associated malaria. The invention also provides immunogenic compositions and vaccines that are useful for preventing malaria in pregnant women.

Abstract: The present invention relates to 4-azapodophyllotoxin analogs of formula (I) in which X, R1, R2, R3, R4 and Ar are as defined in claim 1, preferably a pharmaceutically acceptable salt thereof, optionally in the form of a solvate, a composition comprising said analogs, their use as medicament, in particular for the treatment of cancer, and a process for their preparation.

Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring 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.

Abstract: The invention concerns decorin for increasing muscle mass, particularly in the treatment of muscular dystrophies.

Abstract: The invention relates to the use of fibromodulin and lumican, particularly active fragments thereof, to increase muscle mass, especially in the treatment of muscular dystrophies.

Abstract: 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.

Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring 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.

Abstract: Multimodal optical and magnetic resonance imaging methods based on the use of persistent luminescence nanoparticles. Use of mesoporous persistent luminescence <<core-shell>> complexes for theranostic applications.