Current Gene Therapy - Volume 11, Issue 2, 2011

The better the understanding of cellular and molecular events involved in nervous system degeneration and regeneration became during the last decades, the more likely have gene therapy approaches become to improve nervous system regeneration and to find their position in clinical settings. Gene therapy based strategies for neuroregeneration mainly aim to provide target specific neurotrophic support to enhance vi Read More

Peripheral nerve injury in humans often leads to incomplete functional recovery. In this review we discuss the potential for gene therapy to be used as a strategy alongside surgical repair techniques for the study of peripheral nerve regeneration in rodent models and with a view to its eventual use for the promotion of successful regeneration in the clinic. Gene therapy vectors based on herpes simplex virus, adenovirus, lentiviru Read More

The inability of the central nervous system (CNS) to efficiently repair damages results in severe functional impairment after trauma or neurodegenerative / demyelinating diseases. Regeneration failure is attributed to inhibitory molecules creating a nonpermissive environment for axonal regrowth, and dictates the necessity for the development of novel therapeutic strategies. An emerging approach for improving r Read More

Recent understanding in pathophysiological mechanisms of spinal cord and spinal root injuries has facilitated the development of new strategies to promote neural repair. Gene therapy approaches have been viewed as the ideal means to achieve long-term local delivery of therapeutic molecules in the central nervous system (CNS). Ex vivo gene delivery offers the additional advantage of providing cellular support for re Read More

Recent clinical trials have shown that the use of replication deficient viral vectors to genetically modify cells in the retina can be of therapeutic benefit in the treatment of certain inherited degenerative conditions that compromise photoreceptor, and hence visual, function. This review is focussed primarily on the use of recombinant adeno-associated viral (rAAV) vectors to target neurons in inner retina, specifically retinal Read More

Intravenous enzyme replacement therapy has been developed as a viable treatment for most of the somatic pathologies associated with the mucopolysaccharide storage disorders. However, approximately two thirds of individuals affected by a mucopolysaccharide storage disorder also display neurological disease, in these instances intravenous enzyme replacement therapy is not viable as the blood-brain barrier severel Read More

Classical non-viral methods of gene transfer, such as chemical transfection, have met with limited success of instillation of genetic material into non-proliferating cells in vitro. Among the different kinds of viral vectors, Lentiviral vectors (LVs) have emerged as robust and versatile tool for ex vivo and in vivo gene delivery into multiple cell types including non-dividing cells such as neurons. The capacity of LVs to maintain stable, Read More