Volume 12 Issue 5, May 2011

Cell-based therapy is a growing field that exploits advances in stem cell manipulation and gene transfer technologies. This Review discusses the latest developments and crucial challenges for this field, with an emphasis on haematopoietic stem cell gene therapy.

This Review describes the state-of-the-art accomplishments that have been made with gene-based therapies and the major barriers that need to be overcome before these tools are more widely implemented by the medical community.

RNA interference can elicit specific gene silencing and so holds great potential for treating infectious or genetic diseases. Several small-RNA-based therapies have now reached clinical trials, but further work is still needed to improve delivery and efficacy.

Recent clinical and preclinical studies have described exciting results using recombinant adeno-associated virus (AAV) vectors for therapeutic gene transfer in genetic disease. This Review explores the potential for using this form of therapy in the context of different tissues and diseases.

Genetic and genomic approaches — including high-throughput sequence analysis and transcriptomics experiments — are revealing a clearer picture of the pathophysiological steps underlying the different forms of heart failure (genetic and acquired) and the genomic responses to cardiac overload.

Structural variation in the genome can influence disease, complex traits and evolution, but comprehensive characterization of variants is challenging. This Review compares current methods — particularly microarray platforms and sequencing-based computational analysis — and considers future research strategies.

Delivering genes or RNAs to cells and tissues, or correcting existing genes, is an approach with great potential to treat a range of diseases. Since its inception about 20 years ago, the field of gene-based therapies has met with considerable challenges and controversy. However, recent progress has helped to realize the initial promise of these approaches and several gene-based therapies have now achieved success in clinical trials.