DEVELOPMENT OF A GENE THERAPY
FOR COCKAYNE SYNDROME TYPE B
What is Cockayne Syndrome Type B?
Cockayne Syndrome (CS) is a multisystem rare disorder characterized by different clinical manifestations resembling accelerated aging, such as severe neurological impairments, pigmentary retinopathy, speech and muscle contraction, and deafness, among others. CS is an infantile disease with a prevalence of 2.7 per 1 million births in western Europe, resulting in premature death by 12 years of age on average.
The CureCSB Project
The CureCSB project aims to develop a gene therapy by using viral vectors as carriers of the genetic material.
Gene therapy is an approach used to substitute a defective gene through delivery of a functional gene to the target cells.
The goal of CureCSB project is to develop a functional viral vector carrying the CSB gene and test it in CSB disease models with the purpose of translating this gene therapy to the clinical and bring a new future for CSB patients.
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