Seminario 2

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Santé & Médecine Technologie

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

INTRODUCTION ,[object Object],[object Object]

PRO051 ( antisense oligonucleotides or molecular patch). ,[object Object]

Recognizes specific sequences of mRNA and the splicing pattern changes and leads to the restoration of the open reading frame.,[object Object]

GENERAL OBJECTIVE ,[object Object],[object Object]

Detecta la unión antígeno-anticuerpo por actividad enzimática y analiza la cantidad de proteína.

Se utiliza para determinar la expresión de genes en diversos tejidos.,[object Object]

Dosis : 0.5 mg, 2mg, 4mg, 6mg/kg de peso.

Biopsias musculares( tibial anterior) 2 semanas después del tratamiento con PRO051.

SEGURIDAD Y TOLERABILIDAD ,[object Object],Niveles aspartatoaminotransferasa. Niveles alaninaaminotransferasa. ,[object Object],Supervisión en orina de alfa1-microglobulina y proteinuria.

Análisis de muestras de suero 120 días antes y después del tratamiento.

Detección de anticuerpos inmunoglobulina GContra distrofina.

FARMACOCINÉTICA ,[object Object], Alcanza niveles máximos a las 2-3 horas. Los niveles incrementan con el # de inyecciones Vida media en plasma de 29 días

RESULTADOS Deleción de los exones 45 a 50

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

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