Silencing of frataxin gene expression triggers p53-dependent apoptosis in human neuron-like cells

Hum. Mol. Genet. (2011) doi: 10.1093/hmg/ddr187

Gloria M. Palomo 1,2, Toñi Cerrato 1,2, Ricardo Gargini1 and Javier Díaz-Nido 1,2

1 Departamento de Biología Molecular and Centro de Biología Molecular “Severo Ochoa” (UAM-CSIC), C/ Nicolás Cabrera, 1. Campus de Cantoblanco. Universidad Autónoma de Madrid. 28049 Madrid, Spain
2 CIBER de Enfermedades Raras (CIBERER). 28049 Madrid, Spain

Keywords: Friedreich’s ataxia, frataxin, neuronal cells, lentiviral vectors, minigenes, frataxin-specific shRNAs, human neuroblastoma SH-SY5Y cells, p53, PUMA, Bax, caspase-3, autophagy, useful cell model, neurodegeneration.

European regulation on orphan medicinal products: 10 years of experience and future perspectives

Nature Reviews Drug Discovery 10, 341-349 (May 2011) | doi:10.1038/nrd3445

The Committee for Orphan Medicinal Products and the European Medicines Agency. Scientific Secretariat ¹

  

Keywords: orphan medicinal products,  European Union,  rare conditions,  orphan drug designations, Committee for Orphan Medicinal Products (COMP),  marketing authorization in Europe.