Estrategias de transferencia génica en cerebro mediadas por vectores virales adenoasociados

  1. José Miguel Brito Armas
  2. Ibrahim González Marrero
  3. Agustín Lorenzo Castañeyra Perdomo
  4. Emilia María Carmona Calero
  5. Antonio Alayón Fumero
  6. Javier R. Castro Hernández
  7. Rafael Castro Fuentes
Journal:
Majorensis: Revista Electrónica de Ciencia y Tecnología

ISSN: 1697-5529

Year of publication: 2009

Issue: 5

Pages: 1-13

Type: Article

Export: RIS

Abstract

The expression in the brain of exogenously administered genes has been proposed as an alternative therapy for a large variety of hereditary and acquired diseases of the SNC, and in particular for those whose available treatment does not offer complete recovery and can even have serious side effects. There has been much research into gene therapy recently, with significant progress having been made in the development of new techniques of not only viral but also non viral gene delivery in the SNC, and in the evaluation of its potential for treating neurodegenerative diseases. In the last ten years, viral gene transfer has progressed from merely being an application in animal research to becoming an experimental therapeutic technique in humans. Adeno-associated viral vectors have been one of the most widely used of the many different viral systems developed for this purpose. The degree of gene expression and its distribution in the brain are going to depend on the structure of the vector, on the promoters used, and on the site, volume and rate of injection. However, due to the difficulty which the gene medicines have in crossing the blood brain barrier, invasive administration routes are being used which produce a localized gene expression , despite the fact that many neurodegenerative disorders require transgene delivery to the whole of the SNC. This review looks at the different gene delivery techniques , via adeno-associated viral vectors, which are being developed with therapeutic aims for brain diseases.