Resumen

RAFAEL, Castro. Brain gene therapy: triumph and future of nanotechnology. Rev. iberoam. cienc. tecnol. soc. [online]. 2012, vol.7, n.20, pp.111-128. ISSN 1850-0013.

Brain gene therapy involves the input of nucleic acids into nerve tissue for therapeutic purposes. The genetic material is indirectly introduced into the blood through non-invasive gene therapy, thereby avoiding direct injection into the brain which can damage the blood-brain barrier. Such therapy offers exciting new treatments for the numerous neurological diseases which lack effective pharmacological treatments. In recent years there has been a dramatic shift in non- invasive strategies for transferring genes into the central nervous system. The development of  new serotypes of adenoassociated vectors and of a range of functionalized nanoparticles means that it is now possible to introduce and express gene material in nerve tissue following peripheral administration of the vectors mentioned above. Studies already performed on animals have had highly promising results and it is likely that over the next few years they will give rise to non-invasive gene therapy procedures which will be useful and safe for treating patients. The future of gene therapy nanotechnology will open the development of new materials and formation of hybrid vectors that improve efficiency and selectivity while maintaining the equilibrium that must exist between human needs and scientific-technological innovation.

Palabras clave : Gene therapy; Nanoparticles; Brain; AAV9; Society.

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