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CIBER-BBN » Annual report 2014
molecules or functional groups, forming a biocompatible coating . In vitro assays have shown that both types of particle functionalized or not, are not cytotoxic . These studies also shown that these particles can be efficiently internalized into the cells, through interaction with specific membrane receptors . The viability rate of the cells, in the presence of these particles, was 95-98%, after 72 hours in culture . This invention also provides pharmaceutical formulations including active biological molecules in these particles .
MESENCHYMAL CELLS AND A COMPOUND MEMBRANE FOR THE TREATMENT OF OSTEOCHONDRAL LESIONS
Spanish Patent ES2380674 B1
The present invention provides and efficient product for the cellular therapy of articular cartilage lesions, which is prepared in vitro, and is based in autologous adult mesenchymal stem cells and a biomaterial with a new fibrillar organization in the shape of a compound membrane .
Thanks to this product, in the chondro-osteo-articular lesion a regenerative tissue is formed equal to the adjacent cartilage in the three cartilaginous strata, and equal to the subchondral bone in the organization of the cells, and in the amount and quality of either the cartilaginous or the bone surrounding matrix . The generated tissue integrates in a permanent way in the receptor tissue, and, besides, it is functional regarding its response to loads .
The authors of the present invention have demonstrated that this product allows the formation of cartilage in a natural way, therefore the new tissue is formed de novo, and integrates in the treated tissue, allowing a stable, long-lasting, and functional regeneration . That is, the product allows a intra- tissue, rather than a lesion, repair .
Therefore, a first aspect of the present invention refers to a composition comprising mesenchymal stem cells and a compound membrane . The term “compound” make reference to that the membrane presents at least two layers with different structure . A second aspect of the present invention refers to the use of the pharmaceutical composition to prepare a drug for the treatment of a cartilage lesion, for the regeneration of the cartilage, for the treatment of a bone lesion, for the regeneration of the bone, for the treatment of a osteochondral lesion, for the treatment of osteoarthritis, for the regeneration of the cartilage . A third aspect of the present invention refers to an obtaining method for the pharmaceutical composition of the invention .
MODIFIED OLIGONUCLEOTIDES AS REGULATORS OF GENE EXPRESSION
Spanish Patent ES2372237B1
In the present invention, new siRNA compounds are described, which are more stable and efficient in inhibiting gene expression . The synthesis procedures for oligoribonucleotides containing chemical groups are also described .
The presence of these groups improves the thermodynamic stability of the siRNA duplex . These duplexes can transfect human cells, and the conjugates efficiently enter in the RNA mechanism of interference, in a similar way to the unmodified ones, inducing specific inhibition of the gene with the complementary sequence to the siRNA . Besides, the modified siRNA have a higher stability to nucleases present in serum than the unmodified ones . Therefore, those described in this invention can keep the gene silencing for a longer time than those unmodified . Another aspect is the pharmaceutical compositions containing these siRNA compounds and, at least, an excipient or pharmaceutical vehicle, and the use of these compounds or their pharmaceutical compositions for the preparation of drugs .
The excipients include any inert material used for the preparation of a composition for pharmaceutical dosage . A particular excipient is the transfection agents that increase the vector properties of the composition . The preferred administration of the pharmaceutical compositions of this invention is oral or parenteral .