Current Gene Therapy - Volume 7, Issue 3, 2007

Gene transfer into hematopoietic stem cells has been successfully used to correct immunodeficiencies affecting the lymphoid compartment. However, similar results have not been reported for diseases affecting myeloid cells, mainly due to low engraftment levels of gene-modified cells observed in unconditioned patients. Here we review the developments leading to a gene therapy approach for the treatment of Chronic Read More

The introduction of genes through the skin has been an attractive and dynamic field of research in recent years. It gives the first gleam of hope in therapy for the human genetic diseases that mainly affect this tissue, such as patients that suffer from xeroderma pigmentosum, and who experience increased frequency of skin cancer. The first in vitro experiments were successful in correcting the genetic defects of cells fr Read More

Cystic fibrosis (CF) is caused by mutations of the CF transmembrane conductance regulator (CFTR) gene, which encodes a cAMP dependent chloride channel whose expression is finely tuned in space and time. Gene therapy approaches to CF lung disease have demonstrated partial efficacy and short-lived CFTR expression in the airways. Drawbacks in the use of classical gene transfer vectors include immune respons Read More

Gene delivery vectors based on Adenoviral (Ad) vectors have enormous potential for the treatment of both hereditary and acquired disease. Detailed structural analysis of the Ad virion, combined with functional studies has broadened our knowledge of the structure/function relationships between Ad vectors and host cells/tissues and substantial achievement has been made towards a thorough understanding of the biology Read More

The development of gene-inactivation systems is an active and important field for both functional genomics and gene therapy. Towards this end, ribozymes (i.e. RNA enzymes), that specifically recognize and subsequently catalyze the cleavage of other target RNA molecules, are attractive molecular tools. Ribozymes represent an interesting alternative to the RNA interference (RNAi) approach for gene inactivation, especiall Read More

Wilson disease is a rare autosomal-recessive copper overload disorder due to mutations of the Wilson disease gene ATP7B. The disease typically manifests at late childhood or in young adults with hepatic and/or neurological symptoms. Being fatal without medical treatment or liver transplantation the long-term outcome of Wilson disease depends on the adherence to an effective treatment. Because current medical Read More

Gene transfer to a transected peripheral nerve or avulsed nerve root is discussed to be helpful where neurosurgical peripheral nerve reconstruction alone will not result in full recovery of function. Axonal regeneration is supposed to be facilitated by this new therapeutic approach via delivery of specific regeneration promoting molecules as well as survival proteins for the injured sensory and motor neurons. Theref Read More