Gene therapy, a technique for correcting defective genes that are responsible for disease development can be used more effectively by targeting gene delivery and high gene expression levels in bone marrow by the use of advanced genetic tools and techniques. This was recently reported with examples in five engaging articles published in the latest issue of Human Gene Therapy.

To select and target any particular immune cell population for cell and gene therapy has become much easier due to modern gene marking techniques as stated by Barese and Dunbar. The authors of the review article “Contributions of Gene Marking to Cell and Gene Therapies” conclude that the favorable outcome of marking studies will enable better optimization in gene transfer for immunotherapeutics, and enhance patient survival.

The use of polymerase chain reaction (PCR) and multiple next-generation methods of DNA sequencing (techniques & technologies that are used for determining the order of the nucleotide bases in a molecule of DNA) to identify various gene products that are linked with successful long-term transfer of therapeutic genes to bone marrow were examined by Giordano et al. Their study results have been published in the article entitled “Clonal Inventory Screens Uncover Monoclonality Following Serial Transplantation of MGMTP140K-Transduced Stem Cells and Dose-Intense Chemotherapy.”

Walia et al. recently conducted a study for the treatment of lysosomal storage disorders in which the therapeutic gene delivery to bone marrow and peripheral blood cells was tested. The study was entitled “Autologous Transplantation of Lentivector/Acid Ceramidase-Transduced Hematopoietic Cells in Nonhuman Primates,” and the results have provided clear evidence that transduction with the AC gene can replace acid ceramidase (AC) gene activity and lead to a reduction of ceramide levels in blood cells.

The article entitled “Gene Therapy for Canine Leukocyte Adhesion Deficiency with Lentiviral Vectors Using the Murine Stem Cell Virus and Human Phosphoglycerate Kinase Promoters” describes the results of a study conducted by Hunter and his colleagues in which the scientists compared the use of a human gene promoter with a mouse promoter-enhancer for obtaining high levels of gene expression in a dog model of leukocyte adhesion deficiency type 1.

David Emery, in the Methods Review entitled “The Use of Chromatin Insulators to Improve the Expression and Safety of Integrating Gene Transfer Vectors,” has stressed upon available evidence that supports the efficient use of chromatin insulators to improve the expression and safety of gene transfer vectors. Chromatin insulators are a class of DNA regulatory elements which have evolved to regulate transcription by using regulatory elements that are often distant from each other on the linear genome.

James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia states:

“Bone marrow-directed gene therapy was the first model considered in the treatment of genetic diseases and remains one of the most successful models in terms of clinical efficacy.”

Source: Human Gene Therapy Written by Anne Hudsmith