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Possible Treatments for Progeria: A Rare Genetic Disorder

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Have you ever come across a child with aged skin, short stature and low body weight? If yes, probably that child must be suffering from a very rare genetic disorder: Progeria. This disease is also called as Hutchinson–Gilford Progeria Syndrome (HGPS). The cause of this disorder is genetic mutations that occur during the fetal development. They live till their mid teens or early twenties. But most of the children with this rare disorder die due to cardiovascular diseases.

Initially there was no cure for this disorder.  A Team from INSERM, led by Nicolas Lévy and Annachiara De Sandre-Giovannoli succeeded in finding an animal model for the disease. The mice that were used for the research with Progeria, when treated with gene therapy survived for long duration than other mice and this model depicted the effects of Progeria. No animal model was able to depict the effects of this disease in humans before this research. Later statins were used for treating cardiovascular risks and aminobisphosphonates for preventing other complications like cancer. But this was not a success as these medicines did not reduce the progerin quantity which is the many cause of Progeria.

The invention of Nanodevice by the Spanish scientists at Centro de Reconocimiento Molecular y Desarrollo Tecnológico (IDM), Mixed Unit Universitat Politècnica de València-Universitat de València; the Instituto de Biología Molecular y Celular de Plantas (UPV-CSIC), the Instituto de Investigaciones Biomédicas (CSIC/UAM), the CIBER of Rare Diseases (CIBERER) and CIBER on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN) gave a very promising treatment in the recent years for treating Progeria. This device is designed with mesoporous nanoparticles with a galacto-oligosaccharide in the outer surface. The aged senescent cells will be reduced thus slowing the process of aging.


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