Strategies for the development of cell lines for ex vivo gene therapy in the central nervous system

Cell Transplant. 2011;20(7):983-1001. doi: 10.3727/096368910X546599. Epub 2010 Dec 22.

Abstract

Disorders of the central nervous system (CNS) as a result of trauma or ischemic or neurodegenerative processes still pose a challenge for modern medicine. Due to the complexity of the CNS, and in spite of the advances in the knowledge of its anatomy, pharmacology, and molecular and cellular biology, treatments for these diseases are still limited. The development of cell lines as a source for transplantation into the damaged CNS (cell therapy), and more recently their genetic modification to favor the expression and delivery of molecules with therapeutic potential (ex vivo gene therapy), are some of the techniques used in search of novel restorative strategies. This article reviews the different approaches that have been used and perfected during the last decade to generate cell lines and their use in experimental models of neuronal damage, and evaluates the prospects of applying these methods to treat CNS disorders.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Cell Line
  • Cellular Reprogramming
  • Central Nervous System Diseases / therapy*
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors / metabolism
  • Humans
  • Neural Stem Cells / cytology
  • Neural Stem Cells / transplantation
  • RNA, Small Interfering / metabolism

Substances

  • RNA, Small Interfering