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

  1. Pignataro López, José Diego
unter der Leitung von:
  1. Gloria González Aseguinolaza Doktorvater/Doktormutter
  2. José Luis Lanciego Pérez Co-Doktorvater/Doktormutter

Universität der Verteidigung: Universidad de Navarra

Fecha de defensa: 20 von April von 2017

Gericht:
  1. José Luis Labandeira-García Präsident
  2. Cristian Smerdou Picazo Sekretär/in
  3. Juan Gerez Vocal
  4. Alberto Rico Santos Vocal
  5. María Rosario Luquin Piudo Vocal

Art: Dissertation

Teseo: 146757 DIALNET lock_openDadun editor

Zusammenfassung

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.