Cell and ex vivo gene therapy: Advances in the treatment of central nervous system disorders [Terapia celular y terapia génica ex vivo: Avances en el tratamiento de enfermedades del sistema nervioso central] Review uri icon

abstract

  • Introduction. The direct application of different types of cells to the central nervous system (CNS) by means of transplants, so-called cell therapy, is an experimental approach that promotes the characterisation of the cell and molecular mechanisms involved in the development, plasticity and regeneration of damage to the CNS. Knowledge of the pathology and aetiology of neurodegenerative diseases, which are frequently related to the neurodegeneration of selected types of cells and/or deficiency of particular neurotransmitters, has led to research on means to obtain cell lines with specific characteristics. Development. In some cases these cells become genetically transformed to produce large amounts of neurotransmitters or neurotrophic factors, the well-known ex vivo gene therapy, so that they can be used as therapeutic alternatives in pathologies affecting the CNS. For example, reports have been published of the beneficial effects of these therapies in studies with humans and in different models of neurodegenerative diseases, such as Huntington's disease and Parkinson's disease, and in epilepsy. The aim of this work is to review the different studies in which transplants of neuronal and non-neuronal cells have been used and which have served to further our knowledge of the CNS, of diseases that affect it and of possible therapeutic alternatives. Conclusions. Ex vivo cell therapy and gene therapy have helped to expand our knowledge about plasticity and the mechanisms and factors that promote cell integration within the central nervous system. Although behavioural improvements have been reported in animal and human models, further work is still required on these studies to clear up a number of dubious points. Ex vivo cell therapy and gene therapy in the nervous system constitute an important methodological tool with therapeutic possibilities that deserve further study. © 2009, Revista de Neurología.

publication date

  • 2009-01-01