SystemicAdministration of PRO051in Duchenne’s Muscular Dystrophy Nathalie M. Goemans, M.D., Mar Tulinius, M.D., Ph.D.,Johanna T. van den Akker, Ph.D., Brigitte E. Burm, Ph.D., Peter F. Ekhart, M.Sc.,Niki Heuvelmans, TjadineHolling, Ph.D., Anneke A. Janson,Gerard J. Platenburg, M.Sc., Jessica A. Sipkens, M.Sc., J.M. Ad Sitsen, M.D., Ph.D.,Annemieke Aartsma-Rus, Ph.D., Gert-Jan B. van Ommen, Ph.D.,GunnarBuyse, M.D., Ph.D., NiklasDarin, M.D., Ph.D.,Jan J. Verschuuren, M.D., Ph.D., Giles V. Campion, M.D.,Sjef J. de Kimpe, Ph.D., and Judith C. van Deutekom, Ph.D María Antonia Velásquez Daniela Vásquez Medicina UPB 2011
observed in this study as the antisense oligonucleotide PRO051 complementary strand acts as the dystrophin gene, affected in DMD on exon 51 specifically blocking the protein transcription erroneous thus reducing the effects of the disease and greatly increasing the expression of the protein dystrophin in muscle fibers of patients, allowing the recovery of the major conditions such as muscle weakness, loss of muscle mass, inability to walk, difficulty breathing.
PRO051 ( antisense oligonucleotides or molecular patch).
Sequence oligonucleotides inducing jump in the exon 51 of defective dystrophin gene, joining a sequence of dystrophin pre-mRNA and masking signals the inclusion of exon used for splicing.
Recognizes specific sequences of mRNA and the splicing pattern changes and leads to the restoration of the open reading frame.
RELATIONS OF THE PRO051 1)Masks the exon 51 that is what is most defective in most patients with DMD, avoiding an erroneous transcription and translation and getting a new dystrophin is synthesized which reduces the effects of the disease. 2) To be a complementary strand of the dystrophin gene, the splicing pattern changes in defective mRNA and restores the reading frame for preventing expression of the gene wrong
CONCLUSIONS The method studied in this project is a new hope of recovery for the persons with Duchenne's Muscular Dystrophy It is an innovative method that though it is not the exit of the disease would provide a well-known improvement in the quality of life of these persons The therapy antisense using oligonucleotides as the PRO051 it is very effective for the treatment of the patients with DMD, since these manage to block the expression of the defective gene of dystrofin, diminishing this way his effects The implementation of this constant treatment with PRO051 manages to increase the life expectation of the patients with DMD