Abstract: The invention relates to the use of 4,6,4?-trimethylangelicin (TMA) and structural analogs thereof to prepare a medicament for the treatment of cystic fibrosis with the primary objective of correcting the defective CFTR in a sub-group of cystic fibrosis patients consisting of patients carrying the F508del-CFTR mutation.

Abstract: The present invention relates to compounds having cytostatic activity against tumor cells. The compounds of the invention are of formula (I), or derivatives hereof, wherein R0, R1, R2, A, and X have defined meanings as described in claim 1.

Abstract: The invention relates to the use of 4,6,4?-trimethylangelicin (TMA) and structural analogues thereof to prepare a medicament for the treatment of cystic fibrosis with the primary objective of correcting the defective CFTR in a sub-group of cystic fibrosis patients consisting of patients carrying the F508de1-CFTR mutation.

Abstract: A modified human U1snRNA molecule is described, the target sequence of which is located in a region of the pre-mRNA of the target gene comprised between 2 and 50 base pairs downstream of an exon/intron junction site, which is capable of restoring the correct splicing of a target gene of therapeutic interest bearing a mutation which induces exon skipping and resulting in a genetic disease. Modified human U1snRNA molecules are described by way of example for the correction of diseases associated with exon skipping, such as spinal muscular atrophy, hemophilia B, and cystic fibrosis.

Abstract: The present invention relates to compounds having cytostatic activity against tumor cells. The compounds of the invention are of formula (I), or derivatives hereof, wherein R0, R1, R2, A, and X have defined meanings as described in claim 1.

Abstract: Compound with structure 2-phenylbenzimidazole of formula I and/or a pharmaceutically acceptable salt thereof, wherein said compound of formula I corresponds to: in which n is 1, 2 or 3 and in which R is a carboxyl radical (—COOH) or a sulphonic radical (—SO3H); pharmaceutical and/or cosmetic formulation and/or medical device including such a compound; method for synthesising it.

Abstract: A modified human U1snRNA molecule is described, the target sequence of which is located in a region of the pre-mRNA of the target gene comprised between 2 and 50 base pairs downstream of an exon/intron junction site, which is capable of restoring the correct splicing of a target gene of therapeutic interest bearing a mutation which induces exon skipping and resulting in a genetic disease. Modified human U1snRNA molecules are described by way of example for the correction of diseases associated with exon skipping, such as spinal muscular atrophy, hemophilia B, and cystic fibrosis.

Abstract: The invention relates to a synthetic double-stranded oligonucleotide capable of modifying the molecular phenotype of osteoclasts and increasing the expression of the oestrogen alpha receptor gene. Pharmaceutical compositions comprising the oligonucleotide according to the invention are also described, as well as therapeutic applications of that oligonucleotide, in particular for the treatment of osteopenic diseases such as for example osteoporosis. The oligonucleotide according to the invention is characterized in that it comprises the sequence 5?-ATTTATTTTCAATACTGACT-3? (SEQ ID NO: 1) or a fragment or a mutant thereof.

Abstract: The use of angelicin and its structural analogues for the preparation of a medicament for the therapeutic treatment of beta-thalassaemia is described. A structural analogue which is particularly preferred for this purpose is bergapten.

Abstract: The invention relates to a synthetic double-stranded oligonucleotide capable of modifying the molecular phenotype of osteoclasts and increasing the expression of the oestrogen alpha receptor gene. Pharmaceutical compositions comprising the oligonucleotide according to the invention are also described, as well as therapeutic applications of that oligonucleotide, in particular for the treatment of osteopenic diseases such as for example osteoporosis. The oligonucleotide according to the invention is characterised in that it comprises the sequence 5?-ATTTATTTTCAATACTGACT-3? or a fragment or a mutant thereof.

Abstract: The invention relates to the use of structural analogues of distamycin having the general formula (I), (II), (III), (IV) or (V) or their pharmaceutically acceptable salts, for the preparation of a medicament for the therapeutic treatment of ?-thalassaemia.

Abstract: The invention refers to a synthetic double-stranded oligonucleotide having a length comprised between 10 and 50 bases and a nucleic acid sequence selected from the group consisting of: (a) sequences corresponding to a selected portion of the promoter of human ?-globin gene; and (b) sequences corresponding to a selected portion of the human genomic region comprised between the ?-globin gene and the ??-cluster, for use as an inducer of erythroid differentiation.