rAAV8-mediated in vivo reprogramming of striatal astrocytes into neurons as a potential therapeutic strategy for neurodegenerative diseases

• Autores: José Diego Pignataro López
• Directores de la Tesis: Gloria González Aseguinolaza (dir. tes.), José Luis Lanciego Pérez (codir. tes.)
• Lectura: En la Universidad de Navarra ( España ) en 2017
• Idioma: inglés
• Número de páginas: 177
• Tribunal Calificador de la Tesis: José Luis Labandeira-García (presid.), Cristian Smerdou Picazo (secret.), Alberto Rico Santos (voc.), María Rosario Luquin Piudo (voc.), Juan Gerez (voc.)
• Programa de doctorado: Programa de Doctorado en Investigación Biomédica por la Universidad de Navarra
• Materias:
  • Ciencias de la vida
    • Biología celular
      • Cultivo celular
    • Genética
      • Ingeniería genética
    • Biología molecular
• Enlaces
  • Tesis en acceso abierto en: Dadun
• Resumen

  • CNS neurodegenerative disorders represent a major health problem worldwide. In PD, the progressive loss of miDA neurons leads to an irreversible dopamine deficiency in the striatum, ultimately triggering the appearance of the cardinal motor symptoms that typically characterize this movement disorder of basal ganglia origin. Accordingly, the so-called in vivo direct reprogramming has recently emerged as an appealing technical choice to further generate functional DA neurons from endogenous glial cells in situ. In this regard, here we are taking advantage of newly-designed adeno-associated viral vectors in an attempt to overexpress a number of transcription factors within striatal astrocytes to compensate the lack of dopamine in this brain region. The main goal of this project is the development of new gene therapy tools to drive gene expression in specific cell populations of the CNS for future applications in neurodegenerative diseases including Parkinson.